DMD Genotypes and Motor Function in Duchenne Muscular Dystrophy: A Multi-institution Meta-analysis With Implications for Clinical Trials

Clinical trials of genotype-targeted treatments in Duchenne muscular dystrophy (DMD) traditionally compare treated patients with untreated patients with the same genotype class. This avoids confounding of drug efficacy by genotype effects but also shrinks the pool of eligible controls, increasing ch...

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Published inNeurology Vol. 100; no. 15; p. e1540
Main Authors Muntoni, Francesco, Signorovitch, James, Sajeev, Gautam, Lane, Henry, Jenkins, Madeline, Dieye, Ibrahima, Ward, Susan J, McDonald, Craig, Goemans, Nathalie, Niks, Erik H, Wong, Brenda, Servais, Laurent, Straub, Volker, Guglieri, Michela, de Groot, Imelda J M, Chesshyre, Mary, Tian, Cuixia, Manzur, Adnan Y, Mercuri, Eugenio, Aartsma-Rus, Annemieke
Format Journal Article
LanguageEnglish
Published United States 11.04.2023
Subjects
Child
Genotype
Humans
Italy
Muscular Dystrophy, Duchenne - drug therapy
Muscular Dystrophy, Duchenne - genetics
Walking
Italy
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Abstract Clinical trials of genotype-targeted treatments in Duchenne muscular dystrophy (DMD) traditionally compare treated patients with untreated patients with the same genotype class. This avoids confounding of drug efficacy by genotype effects but also shrinks the pool of eligible controls, increasing challenges for trial enrollment in this already rare disease. To evaluate the suitability of genotypically unmatched controls in DMD, we quantified effects of genotype class on 1-year changes in motor function endpoints used in clinical trials. More than 1,600 patient-years of follow-up (>700 patients) were studied from 6 real-world/natural history data sources (UZ Leuven, PRO-DMD-01 shared by CureDuchenne, iMDEX, North Star UK, Cincinnati Children's Hospital Medical Center, and the DMD Italian Group), with genotypes classified as amenable to skipping exons 44, 45, 51, or 53, or other skippable, nonsense, and other mutations. Associations between genotype class and 1-year changes in North Star Ambulatory Assessment total score (ΔNSAA) and in 10-m walk/run velocity (Δ10MWR) were studied in each data source with and without adjustment for baseline prognostic factors. The studied genotype classes accounted for approximately 2% of variation in ΔNSAA outcomes after 12 months, whereas other prognostic factors explained >30% of variation in large data sources. Based on a meta-analysis across all data sources, pooled effect estimates for the studied skip-amenable mutation classes were all small in magnitude (<2 units in ΔNSAA total score in 1-year follow up), smaller than clinically important differences in NSAA, and were precisely estimated with standard errors <1 unit after adjusting for nongenotypic prognostic factors. These findings suggest the viability of trial designs incorporating genotypically mixed or unmatched controls for up to 12 months in duration for motor function outcomes, which would ease recruitment challenges and reduce numbers of patients assigned to placebos. Such trial designs, including multigenotype platform trials and hybrid designs, should ensure baseline balance between treatment and control groups for the most important prognostic factors, while accounting for small remaining genotype effects quantified in this study.
AbstractList Clinical trials of genotype-targeted treatments in Duchenne muscular dystrophy (DMD) traditionally compare treated patients with untreated patients with the same genotype class. This avoids confounding of drug efficacy by genotype effects but also shrinks the pool of eligible controls, increasing challenges for trial enrollment in this already rare disease. To evaluate the suitability of genotypically unmatched controls in DMD, we quantified effects of genotype class on 1-year changes in motor function endpoints used in clinical trials. More than 1,600 patient-years of follow-up (>700 patients) were studied from 6 real-world/natural history data sources (UZ Leuven, PRO-DMD-01 shared by CureDuchenne, iMDEX, North Star UK, Cincinnati Children's Hospital Medical Center, and the DMD Italian Group), with genotypes classified as amenable to skipping exons 44, 45, 51, or 53, or other skippable, nonsense, and other mutations. Associations between genotype class and 1-year changes in North Star Ambulatory Assessment total score (ΔNSAA) and in 10-m walk/run velocity (Δ10MWR) were studied in each data source with and without adjustment for baseline prognostic factors. The studied genotype classes accounted for approximately 2% of variation in ΔNSAA outcomes after 12 months, whereas other prognostic factors explained >30% of variation in large data sources. Based on a meta-analysis across all data sources, pooled effect estimates for the studied skip-amenable mutation classes were all small in magnitude (<2 units in ΔNSAA total score in 1-year follow up), smaller than clinically important differences in NSAA, and were precisely estimated with standard errors <1 unit after adjusting for nongenotypic prognostic factors. These findings suggest the viability of trial designs incorporating genotypically mixed or unmatched controls for up to 12 months in duration for motor function outcomes, which would ease recruitment challenges and reduce numbers of patients assigned to placebos. Such trial designs, including multigenotype platform trials and hybrid designs, should ensure baseline balance between treatment and control groups for the most important prognostic factors, while accounting for small remaining genotype effects quantified in this study.
Author Niks, Erik H
Dieye, Ibrahima
Jenkins, Madeline
McDonald, Craig
Goemans, Nathalie
Muntoni, Francesco
Tian, Cuixia
Wong, Brenda
Ward, Susan J
Straub, Volker
Mercuri, Eugenio
Aartsma-Rus, Annemieke
Lane, Henry
Manzur, Adnan Y
Chesshyre, Mary
Signorovitch, James
Guglieri, Michela
Servais, Laurent
de Groot, Imelda J M
Sajeev, Gautam
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  givenname: Francesco
  orcidid: 0000-0002-9102-5232
  surname: Muntoni
  fullname: Muntoni, Francesco
  organization: From the Dubowitz Neuromuscular Centre (F.M., M.C., A.Y.M.), NIHR Great Ormond Street Hospital Biomedical Research Centre, Great Ormond Street Institute of Child Health, University College London, and Great Ormond Street Hospital Trust, United Kingdom; Analysis Group, Inc. (J.S., G.S., H.L., M.J., I.D.), Boston; The Collaborative Trajectory Analysis Project (J.S., S.J.W.), Cambridge, MA; Department of Physical Medicine and Rehabilitation, and Pediatrics (C.M.), University of California, Davis, Sacramento; Child Neurology (N.G.), University Hospitals Leuven, Belgium; Department of Neurology (E.H.N.), Leiden University Medical Centre, the Netherlands; Department of Pediatrics (B.W.), University of Massachusetts Medical School, Worcester; MDUK Oxford Neuromuscular Center (L.S.), Department of Paediatrics, University of Oxford, United Kingdom and Neuromuscular Center of Liège (L.S.), Division of Paediatrics, CHU and University of Liège, Belgium; John Walton Muscular Dystrophy Research Centre (V.S., M.G.), Newcastle University and Newcastle Hospitals NHS Foundation Trust, United Kingdom; Department of Rehabilitation (I.J.M.d.G.), Donders Centre of Neuroscience, Radboud University Nijmegen Medical Center, the Netherlands; Cincinnati Children's Hospital Medical Center (C.T.), and College of Medicine (C.T.), University of Cincinnati, OH; Department of Pediatric Neurology (E.M.), Fondazione Policlinico Gemelli IRCCS, Catholic University, Rome, Italy; and Department of Human Genetics (A.A.-R.), Leiden University Medical Center, the Netherlands
– sequence: 2
  givenname: James
  surname: Signorovitch
  fullname: Signorovitch, James
  email: james.signorovitch@analysisgroup.com
  organization: From the Dubowitz Neuromuscular Centre (F.M., M.C., A.Y.M.), NIHR Great Ormond Street Hospital Biomedical Research Centre, Great Ormond Street Institute of Child Health, University College London, and Great Ormond Street Hospital Trust, United Kingdom; Analysis Group, Inc. (J.S., G.S., H.L., M.J., I.D.), Boston; The Collaborative Trajectory Analysis Project (J.S., S.J.W.), Cambridge, MA; Department of Physical Medicine and Rehabilitation, and Pediatrics (C.M.), University of California, Davis, Sacramento; Child Neurology (N.G.), University Hospitals Leuven, Belgium; Department of Neurology (E.H.N.), Leiden University Medical Centre, the Netherlands; Department of Pediatrics (B.W.), University of Massachusetts Medical School, Worcester; MDUK Oxford Neuromuscular Center (L.S.), Department of Paediatrics, University of Oxford, United Kingdom and Neuromuscular Center of Liège (L.S.), Division of Paediatrics, CHU and University of Liège, Belgium; John Walton Muscular Dystrophy Research Centre (V.S., M.G.), Newcastle University and Newcastle Hospitals NHS Foundation Trust, United Kingdom; Department of Rehabilitation (I.J.M.d.G.), Donders Centre of Neuroscience, Radboud University Nijmegen Medical Center, the Netherlands; Cincinnati Children's Hospital Medical Center (C.T.), and College of Medicine (C.T.), University of Cincinnati, OH; Department of Pediatric Neurology (E.M.), Fondazione Policlinico Gemelli IRCCS, Catholic University, Rome, Italy; and Department of Human Genetics (A.A.-R.), Leiden University Medical Center, the Netherlands. james.signorovitch@analysisgroup.com
– sequence: 3
  givenname: Gautam
  orcidid: 0000-0002-1827-8938
  surname: Sajeev
  fullname: Sajeev, Gautam
  organization: From the Dubowitz Neuromuscular Centre (F.M., M.C., A.Y.M.), NIHR Great Ormond Street Hospital Biomedical Research Centre, Great Ormond Street Institute of Child Health, University College London, and Great Ormond Street Hospital Trust, United Kingdom; Analysis Group, Inc. (J.S., G.S., H.L., M.J., I.D.), Boston; The Collaborative Trajectory Analysis Project (J.S., S.J.W.), Cambridge, MA; Department of Physical Medicine and Rehabilitation, and Pediatrics (C.M.), University of California, Davis, Sacramento; Child Neurology (N.G.), University Hospitals Leuven, Belgium; Department of Neurology (E.H.N.), Leiden University Medical Centre, the Netherlands; Department of Pediatrics (B.W.), University of Massachusetts Medical School, Worcester; MDUK Oxford Neuromuscular Center (L.S.), Department of Paediatrics, University of Oxford, United Kingdom and Neuromuscular Center of Liège (L.S.), Division of Paediatrics, CHU and University of Liège, Belgium; John Walton Muscular Dystrophy Research Centre (V.S., M.G.), Newcastle University and Newcastle Hospitals NHS Foundation Trust, United Kingdom; Department of Rehabilitation (I.J.M.d.G.), Donders Centre of Neuroscience, Radboud University Nijmegen Medical Center, the Netherlands; Cincinnati Children's Hospital Medical Center (C.T.), and College of Medicine (C.T.), University of Cincinnati, OH; Department of Pediatric Neurology (E.M.), Fondazione Policlinico Gemelli IRCCS, Catholic University, Rome, Italy; and Department of Human Genetics (A.A.-R.), Leiden University Medical Center, the Netherlands
– sequence: 4
  givenname: Henry
  surname: Lane
  fullname: Lane, Henry
  organization: From the Dubowitz Neuromuscular Centre (F.M., M.C., A.Y.M.), NIHR Great Ormond Street Hospital Biomedical Research Centre, Great Ormond Street Institute of Child Health, University College London, and Great Ormond Street Hospital Trust, United Kingdom; Analysis Group, Inc. (J.S., G.S., H.L., M.J., I.D.), Boston; The Collaborative Trajectory Analysis Project (J.S., S.J.W.), Cambridge, MA; Department of Physical Medicine and Rehabilitation, and Pediatrics (C.M.), University of California, Davis, Sacramento; Child Neurology (N.G.), University Hospitals Leuven, Belgium; Department of Neurology (E.H.N.), Leiden University Medical Centre, the Netherlands; Department of Pediatrics (B.W.), University of Massachusetts Medical School, Worcester; MDUK Oxford Neuromuscular Center (L.S.), Department of Paediatrics, University of Oxford, United Kingdom and Neuromuscular Center of Liège (L.S.), Division of Paediatrics, CHU and University of Liège, Belgium; John Walton Muscular Dystrophy Research Centre (V.S., M.G.), Newcastle University and Newcastle Hospitals NHS Foundation Trust, United Kingdom; Department of Rehabilitation (I.J.M.d.G.), Donders Centre of Neuroscience, Radboud University Nijmegen Medical Center, the Netherlands; Cincinnati Children's Hospital Medical Center (C.T.), and College of Medicine (C.T.), University of Cincinnati, OH; Department of Pediatric Neurology (E.M.), Fondazione Policlinico Gemelli IRCCS, Catholic University, Rome, Italy; and Department of Human Genetics (A.A.-R.), Leiden University Medical Center, the Netherlands
– sequence: 5
  givenname: Madeline
  surname: Jenkins
  fullname: Jenkins, Madeline
  organization: From the Dubowitz Neuromuscular Centre (F.M., M.C., A.Y.M.), NIHR Great Ormond Street Hospital Biomedical Research Centre, Great Ormond Street Institute of Child Health, University College London, and Great Ormond Street Hospital Trust, United Kingdom; Analysis Group, Inc. (J.S., G.S., H.L., M.J., I.D.), Boston; The Collaborative Trajectory Analysis Project (J.S., S.J.W.), Cambridge, MA; Department of Physical Medicine and Rehabilitation, and Pediatrics (C.M.), University of California, Davis, Sacramento; Child Neurology (N.G.), University Hospitals Leuven, Belgium; Department of Neurology (E.H.N.), Leiden University Medical Centre, the Netherlands; Department of Pediatrics (B.W.), University of Massachusetts Medical School, Worcester; MDUK Oxford Neuromuscular Center (L.S.), Department of Paediatrics, University of Oxford, United Kingdom and Neuromuscular Center of Liège (L.S.), Division of Paediatrics, CHU and University of Liège, Belgium; John Walton Muscular Dystrophy Research Centre (V.S., M.G.), Newcastle University and Newcastle Hospitals NHS Foundation Trust, United Kingdom; Department of Rehabilitation (I.J.M.d.G.), Donders Centre of Neuroscience, Radboud University Nijmegen Medical Center, the Netherlands; Cincinnati Children's Hospital Medical Center (C.T.), and College of Medicine (C.T.), University of Cincinnati, OH; Department of Pediatric Neurology (E.M.), Fondazione Policlinico Gemelli IRCCS, Catholic University, Rome, Italy; and Department of Human Genetics (A.A.-R.), Leiden University Medical Center, the Netherlands
– sequence: 6
  givenname: Ibrahima
  surname: Dieye
  fullname: Dieye, Ibrahima
  organization: From the Dubowitz Neuromuscular Centre (F.M., M.C., A.Y.M.), NIHR Great Ormond Street Hospital Biomedical Research Centre, Great Ormond Street Institute of Child Health, University College London, and Great Ormond Street Hospital Trust, United Kingdom; Analysis Group, Inc. (J.S., G.S., H.L., M.J., I.D.), Boston; The Collaborative Trajectory Analysis Project (J.S., S.J.W.), Cambridge, MA; Department of Physical Medicine and Rehabilitation, and Pediatrics (C.M.), University of California, Davis, Sacramento; Child Neurology (N.G.), University Hospitals Leuven, Belgium; Department of Neurology (E.H.N.), Leiden University Medical Centre, the Netherlands; Department of Pediatrics (B.W.), University of Massachusetts Medical School, Worcester; MDUK Oxford Neuromuscular Center (L.S.), Department of Paediatrics, University of Oxford, United Kingdom and Neuromuscular Center of Liège (L.S.), Division of Paediatrics, CHU and University of Liège, Belgium; John Walton Muscular Dystrophy Research Centre (V.S., M.G.), Newcastle University and Newcastle Hospitals NHS Foundation Trust, United Kingdom; Department of Rehabilitation (I.J.M.d.G.), Donders Centre of Neuroscience, Radboud University Nijmegen Medical Center, the Netherlands; Cincinnati Children's Hospital Medical Center (C.T.), and College of Medicine (C.T.), University of Cincinnati, OH; Department of Pediatric Neurology (E.M.), Fondazione Policlinico Gemelli IRCCS, Catholic University, Rome, Italy; and Department of Human Genetics (A.A.-R.), Leiden University Medical Center, the Netherlands
– sequence: 7
  givenname: Susan J
  surname: Ward
  fullname: Ward, Susan J
  organization: From the Dubowitz Neuromuscular Centre (F.M., M.C., A.Y.M.), NIHR Great Ormond Street Hospital Biomedical Research Centre, Great Ormond Street Institute of Child Health, University College London, and Great Ormond Street Hospital Trust, United Kingdom; Analysis Group, Inc. (J.S., G.S., H.L., M.J., I.D.), Boston; The Collaborative Trajectory Analysis Project (J.S., S.J.W.), Cambridge, MA; Department of Physical Medicine and Rehabilitation, and Pediatrics (C.M.), University of California, Davis, Sacramento; Child Neurology (N.G.), University Hospitals Leuven, Belgium; Department of Neurology (E.H.N.), Leiden University Medical Centre, the Netherlands; Department of Pediatrics (B.W.), University of Massachusetts Medical School, Worcester; MDUK Oxford Neuromuscular Center (L.S.), Department of Paediatrics, University of Oxford, United Kingdom and Neuromuscular Center of Liège (L.S.), Division of Paediatrics, CHU and University of Liège, Belgium; John Walton Muscular Dystrophy Research Centre (V.S., M.G.), Newcastle University and Newcastle Hospitals NHS Foundation Trust, United Kingdom; Department of Rehabilitation (I.J.M.d.G.), Donders Centre of Neuroscience, Radboud University Nijmegen Medical Center, the Netherlands; Cincinnati Children's Hospital Medical Center (C.T.), and College of Medicine (C.T.), University of Cincinnati, OH; Department of Pediatric Neurology (E.M.), Fondazione Policlinico Gemelli IRCCS, Catholic University, Rome, Italy; and Department of Human Genetics (A.A.-R.), Leiden University Medical Center, the Netherlands
– sequence: 8
  givenname: Craig
  surname: McDonald
  fullname: McDonald, Craig
  organization: From the Dubowitz Neuromuscular Centre (F.M., M.C., A.Y.M.), NIHR Great Ormond Street Hospital Biomedical Research Centre, Great Ormond Street Institute of Child Health, University College London, and Great Ormond Street Hospital Trust, United Kingdom; Analysis Group, Inc. (J.S., G.S., H.L., M.J., I.D.), Boston; The Collaborative Trajectory Analysis Project (J.S., S.J.W.), Cambridge, MA; Department of Physical Medicine and Rehabilitation, and Pediatrics (C.M.), University of California, Davis, Sacramento; Child Neurology (N.G.), University Hospitals Leuven, Belgium; Department of Neurology (E.H.N.), Leiden University Medical Centre, the Netherlands; Department of Pediatrics (B.W.), University of Massachusetts Medical School, Worcester; MDUK Oxford Neuromuscular Center (L.S.), Department of Paediatrics, University of Oxford, United Kingdom and Neuromuscular Center of Liège (L.S.), Division of Paediatrics, CHU and University of Liège, Belgium; John Walton Muscular Dystrophy Research Centre (V.S., M.G.), Newcastle University and Newcastle Hospitals NHS Foundation Trust, United Kingdom; Department of Rehabilitation (I.J.M.d.G.), Donders Centre of Neuroscience, Radboud University Nijmegen Medical Center, the Netherlands; Cincinnati Children's Hospital Medical Center (C.T.), and College of Medicine (C.T.), University of Cincinnati, OH; Department of Pediatric Neurology (E.M.), Fondazione Policlinico Gemelli IRCCS, Catholic University, Rome, Italy; and Department of Human Genetics (A.A.-R.), Leiden University Medical Center, the Netherlands
– sequence: 9
  givenname: Nathalie
  orcidid: 0000-0002-5157-7155
  surname: Goemans
  fullname: Goemans, Nathalie
  organization: From the Dubowitz Neuromuscular Centre (F.M., M.C., A.Y.M.), NIHR Great Ormond Street Hospital Biomedical Research Centre, Great Ormond Street Institute of Child Health, University College London, and Great Ormond Street Hospital Trust, United Kingdom; Analysis Group, Inc. (J.S., G.S., H.L., M.J., I.D.), Boston; The Collaborative Trajectory Analysis Project (J.S., S.J.W.), Cambridge, MA; Department of Physical Medicine and Rehabilitation, and Pediatrics (C.M.), University of California, Davis, Sacramento; Child Neurology (N.G.), University Hospitals Leuven, Belgium; Department of Neurology (E.H.N.), Leiden University Medical Centre, the Netherlands; Department of Pediatrics (B.W.), University of Massachusetts Medical School, Worcester; MDUK Oxford Neuromuscular Center (L.S.), Department of Paediatrics, University of Oxford, United Kingdom and Neuromuscular Center of Liège (L.S.), Division of Paediatrics, CHU and University of Liège, Belgium; John Walton Muscular Dystrophy Research Centre (V.S., M.G.), Newcastle University and Newcastle Hospitals NHS Foundation Trust, United Kingdom; Department of Rehabilitation (I.J.M.d.G.), Donders Centre of Neuroscience, Radboud University Nijmegen Medical Center, the Netherlands; Cincinnati Children's Hospital Medical Center (C.T.), and College of Medicine (C.T.), University of Cincinnati, OH; Department of Pediatric Neurology (E.M.), Fondazione Policlinico Gemelli IRCCS, Catholic University, Rome, Italy; and Department of Human Genetics (A.A.-R.), Leiden University Medical Center, the Netherlands
– sequence: 10
  givenname: Erik H
  orcidid: 0000-0001-5892-5143
  surname: Niks
  fullname: Niks, Erik H
  organization: From the Dubowitz Neuromuscular Centre (F.M., M.C., A.Y.M.), NIHR Great Ormond Street Hospital Biomedical Research Centre, Great Ormond Street Institute of Child Health, University College London, and Great Ormond Street Hospital Trust, United Kingdom; Analysis Group, Inc. (J.S., G.S., H.L., M.J., I.D.), Boston; The Collaborative Trajectory Analysis Project (J.S., S.J.W.), Cambridge, MA; Department of Physical Medicine and Rehabilitation, and Pediatrics (C.M.), University of California, Davis, Sacramento; Child Neurology (N.G.), University Hospitals Leuven, Belgium; Department of Neurology (E.H.N.), Leiden University Medical Centre, the Netherlands; Department of Pediatrics (B.W.), University of Massachusetts Medical School, Worcester; MDUK Oxford Neuromuscular Center (L.S.), Department of Paediatrics, University of Oxford, United Kingdom and Neuromuscular Center of Liège (L.S.), Division of Paediatrics, CHU and University of Liège, Belgium; John Walton Muscular Dystrophy Research Centre (V.S., M.G.), Newcastle University and Newcastle Hospitals NHS Foundation Trust, United Kingdom; Department of Rehabilitation (I.J.M.d.G.), Donders Centre of Neuroscience, Radboud University Nijmegen Medical Center, the Netherlands; Cincinnati Children's Hospital Medical Center (C.T.), and College of Medicine (C.T.), University of Cincinnati, OH; Department of Pediatric Neurology (E.M.), Fondazione Policlinico Gemelli IRCCS, Catholic University, Rome, Italy; and Department of Human Genetics (A.A.-R.), Leiden University Medical Center, the Netherlands
– sequence: 11
  givenname: Brenda
  surname: Wong
  fullname: Wong, Brenda
  organization: From the Dubowitz Neuromuscular Centre (F.M., M.C., A.Y.M.), NIHR Great Ormond Street Hospital Biomedical Research Centre, Great Ormond Street Institute of Child Health, University College London, and Great Ormond Street Hospital Trust, United Kingdom; Analysis Group, Inc. (J.S., G.S., H.L., M.J., I.D.), Boston; The Collaborative Trajectory Analysis Project (J.S., S.J.W.), Cambridge, MA; Department of Physical Medicine and Rehabilitation, and Pediatrics (C.M.), University of California, Davis, Sacramento; Child Neurology (N.G.), University Hospitals Leuven, Belgium; Department of Neurology (E.H.N.), Leiden University Medical Centre, the Netherlands; Department of Pediatrics (B.W.), University of Massachusetts Medical School, Worcester; MDUK Oxford Neuromuscular Center (L.S.), Department of Paediatrics, University of Oxford, United Kingdom and Neuromuscular Center of Liège (L.S.), Division of Paediatrics, CHU and University of Liège, Belgium; John Walton Muscular Dystrophy Research Centre (V.S., M.G.), Newcastle University and Newcastle Hospitals NHS Foundation Trust, United Kingdom; Department of Rehabilitation (I.J.M.d.G.), Donders Centre of Neuroscience, Radboud University Nijmegen Medical Center, the Netherlands; Cincinnati Children's Hospital Medical Center (C.T.), and College of Medicine (C.T.), University of Cincinnati, OH; Department of Pediatric Neurology (E.M.), Fondazione Policlinico Gemelli IRCCS, Catholic University, Rome, Italy; and Department of Human Genetics (A.A.-R.), Leiden University Medical Center, the Netherlands
– sequence: 12
  givenname: Laurent
  surname: Servais
  fullname: Servais, Laurent
  organization: From the Dubowitz Neuromuscular Centre (F.M., M.C., A.Y.M.), NIHR Great Ormond Street Hospital Biomedical Research Centre, Great Ormond Street Institute of Child Health, University College London, and Great Ormond Street Hospital Trust, United Kingdom; Analysis Group, Inc. (J.S., G.S., H.L., M.J., I.D.), Boston; The Collaborative Trajectory Analysis Project (J.S., S.J.W.), Cambridge, MA; Department of Physical Medicine and Rehabilitation, and Pediatrics (C.M.), University of California, Davis, Sacramento; Child Neurology (N.G.), University Hospitals Leuven, Belgium; Department of Neurology (E.H.N.), Leiden University Medical Centre, the Netherlands; Department of Pediatrics (B.W.), University of Massachusetts Medical School, Worcester; MDUK Oxford Neuromuscular Center (L.S.), Department of Paediatrics, University of Oxford, United Kingdom and Neuromuscular Center of Liège (L.S.), Division of Paediatrics, CHU and University of Liège, Belgium; John Walton Muscular Dystrophy Research Centre (V.S., M.G.), Newcastle University and Newcastle Hospitals NHS Foundation Trust, United Kingdom; Department of Rehabilitation (I.J.M.d.G.), Donders Centre of Neuroscience, Radboud University Nijmegen Medical Center, the Netherlands; Cincinnati Children's Hospital Medical Center (C.T.), and College of Medicine (C.T.), University of Cincinnati, OH; Department of Pediatric Neurology (E.M.), Fondazione Policlinico Gemelli IRCCS, Catholic University, Rome, Italy; and Department of Human Genetics (A.A.-R.), Leiden University Medical Center, the Netherlands
– sequence: 13
  givenname: Volker
  orcidid: 0000-0001-9046-3540
  surname: Straub
  fullname: Straub, Volker
  organization: From the Dubowitz Neuromuscular Centre (F.M., M.C., A.Y.M.), NIHR Great Ormond Street Hospital Biomedical Research Centre, Great Ormond Street Institute of Child Health, University College London, and Great Ormond Street Hospital Trust, United Kingdom; Analysis Group, Inc. (J.S., G.S., H.L., M.J., I.D.), Boston; The Collaborative Trajectory Analysis Project (J.S., S.J.W.), Cambridge, MA; Department of Physical Medicine and Rehabilitation, and Pediatrics (C.M.), University of California, Davis, Sacramento; Child Neurology (N.G.), University Hospitals Leuven, Belgium; Department of Neurology (E.H.N.), Leiden University Medical Centre, the Netherlands; Department of Pediatrics (B.W.), University of Massachusetts Medical School, Worcester; MDUK Oxford Neuromuscular Center (L.S.), Department of Paediatrics, University of Oxford, United Kingdom and Neuromuscular Center of Liège (L.S.), Division of Paediatrics, CHU and University of Liège, Belgium; John Walton Muscular Dystrophy Research Centre (V.S., M.G.), Newcastle University and Newcastle Hospitals NHS Foundation Trust, United Kingdom; Department of Rehabilitation (I.J.M.d.G.), Donders Centre of Neuroscience, Radboud University Nijmegen Medical Center, the Netherlands; Cincinnati Children's Hospital Medical Center (C.T.), and College of Medicine (C.T.), University of Cincinnati, OH; Department of Pediatric Neurology (E.M.), Fondazione Policlinico Gemelli IRCCS, Catholic University, Rome, Italy; and Department of Human Genetics (A.A.-R.), Leiden University Medical Center, the Netherlands
– sequence: 14
  givenname: Michela
  surname: Guglieri
  fullname: Guglieri, Michela
  organization: From the Dubowitz Neuromuscular Centre (F.M., M.C., A.Y.M.), NIHR Great Ormond Street Hospital Biomedical Research Centre, Great Ormond Street Institute of Child Health, University College London, and Great Ormond Street Hospital Trust, United Kingdom; Analysis Group, Inc. (J.S., G.S., H.L., M.J., I.D.), Boston; The Collaborative Trajectory Analysis Project (J.S., S.J.W.), Cambridge, MA; Department of Physical Medicine and Rehabilitation, and Pediatrics (C.M.), University of California, Davis, Sacramento; Child Neurology (N.G.), University Hospitals Leuven, Belgium; Department of Neurology (E.H.N.), Leiden University Medical Centre, the Netherlands; Department of Pediatrics (B.W.), University of Massachusetts Medical School, Worcester; MDUK Oxford Neuromuscular Center (L.S.), Department of Paediatrics, University of Oxford, United Kingdom and Neuromuscular Center of Liège (L.S.), Division of Paediatrics, CHU and University of Liège, Belgium; John Walton Muscular Dystrophy Research Centre (V.S., M.G.), Newcastle University and Newcastle Hospitals NHS Foundation Trust, United Kingdom; Department of Rehabilitation (I.J.M.d.G.), Donders Centre of Neuroscience, Radboud University Nijmegen Medical Center, the Netherlands; Cincinnati Children's Hospital Medical Center (C.T.), and College of Medicine (C.T.), University of Cincinnati, OH; Department of Pediatric Neurology (E.M.), Fondazione Policlinico Gemelli IRCCS, Catholic University, Rome, Italy; and Department of Human Genetics (A.A.-R.), Leiden University Medical Center, the Netherlands
– sequence: 15
  givenname: Imelda J M
  surname: de Groot
  fullname: de Groot, Imelda J M
  organization: From the Dubowitz Neuromuscular Centre (F.M., M.C., A.Y.M.), NIHR Great Ormond Street Hospital Biomedical Research Centre, Great Ormond Street Institute of Child Health, University College London, and Great Ormond Street Hospital Trust, United Kingdom; Analysis Group, Inc. (J.S., G.S., H.L., M.J., I.D.), Boston; The Collaborative Trajectory Analysis Project (J.S., S.J.W.), Cambridge, MA; Department of Physical Medicine and Rehabilitation, and Pediatrics (C.M.), University of California, Davis, Sacramento; Child Neurology (N.G.), University Hospitals Leuven, Belgium; Department of Neurology (E.H.N.), Leiden University Medical Centre, the Netherlands; Department of Pediatrics (B.W.), University of Massachusetts Medical School, Worcester; MDUK Oxford Neuromuscular Center (L.S.), Department of Paediatrics, University of Oxford, United Kingdom and Neuromuscular Center of Liège (L.S.), Division of Paediatrics, CHU and University of Liège, Belgium; John Walton Muscular Dystrophy Research Centre (V.S., M.G.), Newcastle University and Newcastle Hospitals NHS Foundation Trust, United Kingdom; Department of Rehabilitation (I.J.M.d.G.), Donders Centre of Neuroscience, Radboud University Nijmegen Medical Center, the Netherlands; Cincinnati Children's Hospital Medical Center (C.T.), and College of Medicine (C.T.), University of Cincinnati, OH; Department of Pediatric Neurology (E.M.), Fondazione Policlinico Gemelli IRCCS, Catholic University, Rome, Italy; and Department of Human Genetics (A.A.-R.), Leiden University Medical Center, the Netherlands
– sequence: 16
  givenname: Mary
  surname: Chesshyre
  fullname: Chesshyre, Mary
  organization: From the Dubowitz Neuromuscular Centre (F.M., M.C., A.Y.M.), NIHR Great Ormond Street Hospital Biomedical Research Centre, Great Ormond Street Institute of Child Health, University College London, and Great Ormond Street Hospital Trust, United Kingdom; Analysis Group, Inc. (J.S., G.S., H.L., M.J., I.D.), Boston; The Collaborative Trajectory Analysis Project (J.S., S.J.W.), Cambridge, MA; Department of Physical Medicine and Rehabilitation, and Pediatrics (C.M.), University of California, Davis, Sacramento; Child Neurology (N.G.), University Hospitals Leuven, Belgium; Department of Neurology (E.H.N.), Leiden University Medical Centre, the Netherlands; Department of Pediatrics (B.W.), University of Massachusetts Medical School, Worcester; MDUK Oxford Neuromuscular Center (L.S.), Department of Paediatrics, University of Oxford, United Kingdom and Neuromuscular Center of Liège (L.S.), Division of Paediatrics, CHU and University of Liège, Belgium; John Walton Muscular Dystrophy Research Centre (V.S., M.G.), Newcastle University and Newcastle Hospitals NHS Foundation Trust, United Kingdom; Department of Rehabilitation (I.J.M.d.G.), Donders Centre of Neuroscience, Radboud University Nijmegen Medical Center, the Netherlands; Cincinnati Children's Hospital Medical Center (C.T.), and College of Medicine (C.T.), University of Cincinnati, OH; Department of Pediatric Neurology (E.M.), Fondazione Policlinico Gemelli IRCCS, Catholic University, Rome, Italy; and Department of Human Genetics (A.A.-R.), Leiden University Medical Center, the Netherlands
– sequence: 17
  givenname: Cuixia
  surname: Tian
  fullname: Tian, Cuixia
  organization: From the Dubowitz Neuromuscular Centre (F.M., M.C., A.Y.M.), NIHR Great Ormond Street Hospital Biomedical Research Centre, Great Ormond Street Institute of Child Health, University College London, and Great Ormond Street Hospital Trust, United Kingdom; Analysis Group, Inc. (J.S., G.S., H.L., M.J., I.D.), Boston; The Collaborative Trajectory Analysis Project (J.S., S.J.W.), Cambridge, MA; Department of Physical Medicine and Rehabilitation, and Pediatrics (C.M.), University of California, Davis, Sacramento; Child Neurology (N.G.), University Hospitals Leuven, Belgium; Department of Neurology (E.H.N.), Leiden University Medical Centre, the Netherlands; Department of Pediatrics (B.W.), University of Massachusetts Medical School, Worcester; MDUK Oxford Neuromuscular Center (L.S.), Department of Paediatrics, University of Oxford, United Kingdom and Neuromuscular Center of Liège (L.S.), Division of Paediatrics, CHU and University of Liège, Belgium; John Walton Muscular Dystrophy Research Centre (V.S., M.G.), Newcastle University and Newcastle Hospitals NHS Foundation Trust, United Kingdom; Department of Rehabilitation (I.J.M.d.G.), Donders Centre of Neuroscience, Radboud University Nijmegen Medical Center, the Netherlands; Cincinnati Children's Hospital Medical Center (C.T.), and College of Medicine (C.T.), University of Cincinnati, OH; Department of Pediatric Neurology (E.M.), Fondazione Policlinico Gemelli IRCCS, Catholic University, Rome, Italy; and Department of Human Genetics (A.A.-R.), Leiden University Medical Center, the Netherlands
– sequence: 18
  givenname: Adnan Y
  surname: Manzur
  fullname: Manzur, Adnan Y
  organization: From the Dubowitz Neuromuscular Centre (F.M., M.C., A.Y.M.), NIHR Great Ormond Street Hospital Biomedical Research Centre, Great Ormond Street Institute of Child Health, University College London, and Great Ormond Street Hospital Trust, United Kingdom; Analysis Group, Inc. (J.S., G.S., H.L., M.J., I.D.), Boston; The Collaborative Trajectory Analysis Project (J.S., S.J.W.), Cambridge, MA; Department of Physical Medicine and Rehabilitation, and Pediatrics (C.M.), University of California, Davis, Sacramento; Child Neurology (N.G.), University Hospitals Leuven, Belgium; Department of Neurology (E.H.N.), Leiden University Medical Centre, the Netherlands; Department of Pediatrics (B.W.), University of Massachusetts Medical School, Worcester; MDUK Oxford Neuromuscular Center (L.S.), Department of Paediatrics, University of Oxford, United Kingdom and Neuromuscular Center of Liège (L.S.), Division of Paediatrics, CHU and University of Liège, Belgium; John Walton Muscular Dystrophy Research Centre (V.S., M.G.), Newcastle University and Newcastle Hospitals NHS Foundation Trust, United Kingdom; Department of Rehabilitation (I.J.M.d.G.), Donders Centre of Neuroscience, Radboud University Nijmegen Medical Center, the Netherlands; Cincinnati Children's Hospital Medical Center (C.T.), and College of Medicine (C.T.), University of Cincinnati, OH; Department of Pediatric Neurology (E.M.), Fondazione Policlinico Gemelli IRCCS, Catholic University, Rome, Italy; and Department of Human Genetics (A.A.-R.), Leiden University Medical Center, the Netherlands
– sequence: 19
  givenname: Eugenio
  surname: Mercuri
  fullname: Mercuri, Eugenio
  organization: From the Dubowitz Neuromuscular Centre (F.M., M.C., A.Y.M.), NIHR Great Ormond Street Hospital Biomedical Research Centre, Great Ormond Street Institute of Child Health, University College London, and Great Ormond Street Hospital Trust, United Kingdom; Analysis Group, Inc. (J.S., G.S., H.L., M.J., I.D.), Boston; The Collaborative Trajectory Analysis Project (J.S., S.J.W.), Cambridge, MA; Department of Physical Medicine and Rehabilitation, and Pediatrics (C.M.), University of California, Davis, Sacramento; Child Neurology (N.G.), University Hospitals Leuven, Belgium; Department of Neurology (E.H.N.), Leiden University Medical Centre, the Netherlands; Department of Pediatrics (B.W.), University of Massachusetts Medical School, Worcester; MDUK Oxford Neuromuscular Center (L.S.), Department of Paediatrics, University of Oxford, United Kingdom and Neuromuscular Center of Liège (L.S.), Division of Paediatrics, CHU and University of Liège, Belgium; John Walton Muscular Dystrophy Research Centre (V.S., M.G.), Newcastle University and Newcastle Hospitals NHS Foundation Trust, United Kingdom; Department of Rehabilitation (I.J.M.d.G.), Donders Centre of Neuroscience, Radboud University Nijmegen Medical Center, the Netherlands; Cincinnati Children's Hospital Medical Center (C.T.), and College of Medicine (C.T.), University of Cincinnati, OH; Department of Pediatric Neurology (E.M.), Fondazione Policlinico Gemelli IRCCS, Catholic University, Rome, Italy; and Department of Human Genetics (A.A.-R.), Leiden University Medical Center, the Netherlands
– sequence: 20
  givenname: Annemieke
  surname: Aartsma-Rus
  fullname: Aartsma-Rus, Annemieke
  organization: From the Dubowitz Neuromuscular Centre (F.M., M.C., A.Y.M.), NIHR Great Ormond Street Hospital Biomedical Research Centre, Great Ormond Street Institute of Child Health, University College London, and Great Ormond Street Hospital Trust, United Kingdom; Analysis Group, Inc. (J.S., G.S., H.L., M.J., I.D.), Boston; The Collaborative Trajectory Analysis Project (J.S., S.J.W.), Cambridge, MA; Department of Physical Medicine and Rehabilitation, and Pediatrics (C.M.), University of California, Davis, Sacramento; Child Neurology (N.G.), University Hospitals Leuven, Belgium; Department of Neurology (E.H.N.), Leiden University Medical Centre, the Netherlands; Department of Pediatrics (B.W.), University of Massachusetts Medical School, Worcester; MDUK Oxford Neuromuscular Center (L.S.), Department of Paediatrics, University of Oxford, United Kingdom and Neuromuscular Center of Liège (L.S.), Division of Paediatrics, CHU and University of Liège, Belgium; John Walton Muscular Dystrophy Research Centre (V.S., M.G.), Newcastle University and Newcastle Hospitals NHS Foundation Trust, United Kingdom; Department of Rehabilitation (I.J.M.d.G.), Donders Centre of Neuroscience, Radboud University Nijmegen Medical Center, the Netherlands; Cincinnati Children's Hospital Medical Center (C.T.), and College of Medicine (C.T.), University of Cincinnati, OH; Department of Pediatric Neurology (E.M.), Fondazione Policlinico Gemelli IRCCS, Catholic University, Rome, Italy; and Department of Human Genetics (A.A.-R.), Leiden University Medical Center, the Netherlands
BackLink https://www.ncbi.nlm.nih.gov/pubmed/36725339$$D View this record in MEDLINE/PubMed
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Snippet Clinical trials of genotype-targeted treatments in Duchenne muscular dystrophy (DMD) traditionally compare treated patients with untreated patients with the...
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Genotype
Humans
Italy
Muscular Dystrophy, Duchenne - drug therapy
Muscular Dystrophy, Duchenne - genetics
Walking
Title DMD Genotypes and Motor Function in Duchenne Muscular Dystrophy: A Multi-institution Meta-analysis With Implications for Clinical Trials
URI https://www.ncbi.nlm.nih.gov/pubmed/36725339
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