Single-dose, subcutaneous recombinant phenylalanine ammonia lyase conjugated with polyethylene glycol in adult patients with phenylketonuria: an open-label, multicentre, phase 1 dose-escalation trial
Longo, Nicola, MD, Harding, Cary O, MD, Burton, Barbara K, MD, Grange, Dorothy K, MD, Vockley, Jerry, MD, Wasserstein, Melissa, MD, Rice, Gregory M, MD, Dorenbaum, Alejandro, MD, Neuenburg, Jutta K, MD, Musson, Donald G, PhD, Gu, Zhonghua, PhD, Sile, Saba, MD
Published in The Lancet (British edition) (05.07.2014)
Published in The Lancet (British edition) (05.07.2014)
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Journal Article
Mucopolysaccharidosis Type I Newborn Screening: Best Practices for Diagnosis and Management
Clarke, Lorne A., MD, CM, FRCPC, FCCMG, Atherton, Andrea M., MS, CGC, Burton, Barbara K., MD, FAAP, FACMG, Day-Salvatore, Debra L., MD, PhD, FAAP, FACMG, Kaplan, Paige, MBBCh, FRCPC, FCCMG, FAAP, FACMG, Leslie, Nancy D., MD, Scott, C. Ronald, MD, Stockton, David W., MD, FACMG, Thomas, Janet A., MD, FAAP, FACMG, Muenzer, Joseph, MD, PhD
Published in The Journal of pediatrics (01.03.2017)
Published in The Journal of pediatrics (01.03.2017)
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Journal Article
Efficacy of Sapropterin Dihydrochloride in Increasing Phenylalanine Tolerance in Children with Phenylketonuria: A Phase III, Randomized, Double-Blind, Placebo-Controlled Study
Trefz, Friedrich K., MD, Burton, Barbara K., MD, Longo, Nicola, MD, PhD, Casanova, Mercedes Martinez-Pardo, MD, Gruskin, Daniel J., MD, Dorenbaum, Alex, MD, Kakkis, Emil D., MD, PhD, Crombez, Eric A., MD, Grange, Dorothy K., MD, Harmatz, Paul, MD, Lipson, Mark H., MD, Milanowski, Andrzej, MD, PhD, Randolph, Linda Marie, MD, Vockley, Jerry, MD, PhD, Whitley, Chester B., MD, PhD, Wolff, Jon A., MD, Bebchuk, Judith, ScD, Christ-Schmidt, Heidi, MSE, Hennermann, Julia B., MD
Published in The Journal of pediatrics (01.05.2009)
Published in The Journal of pediatrics (01.05.2009)
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Journal Article
Tetrahydrobiopterin Therapy for Phenylketonuria in Infants and Young Children
Burton, Barbara K., MD, Adams, Darius J., MD, Grange, Dorothy K., MD, Malone, John I., MD, Jurecki, Elaina, MS, RD, Bausell, Heather, RD, LDN, Marra, Kayt D., CDN, Sprietsma, Laurie, RD, LD, Swan, Kathleen T., PhD, RD, CDE
Published in The Journal of pediatrics (01.03.2011)
Published in The Journal of pediatrics (01.03.2011)
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Journal Article
A Cerebrospinal Fluid Collection Study in Pediatric and Adult Patients With Hunter Syndrome
Hendriksz, Christian J., Muenzer, Joseph, Burton, Barbara K., Pan, Luying, Wang, Nan, Naimy, Hicham, Pano, Arian, Barbier, Ann J.
Published in Journal of inborn errors of metabolism and screening (01.12.2015)
Published in Journal of inborn errors of metabolism and screening (01.12.2015)
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Journal Article
Metabolic Cardiomyopathy and Mitochondrial Disorders in the Pediatric Intensive Care Unit
Kane, Jason M., MD, FAAP, Rossi, Janet, MD, FAAP, Tsao, Sabrina, MBBS, MRCP, Burton, Barbara K., MD, FAAP
Published in The Journal of pediatrics (01.11.2007)
Published in The Journal of pediatrics (01.11.2007)
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Journal Article
Conference Proceeding
Expert Opinions on Managing Fertility and Pregnancy in Patients With Mucopolysaccharidosis
Stewart, Fiona, Bentley, Andrew, Burton, Barbara K, Guffon, Nathalie, Hale, Susan L., Harmatz, Paul R., Kircher, Susanne G., Kochhar, Pavan K., Mitchell, John J., Plöckinger, Ursula, Semotok, Jennifer, Graham, Sue, Sande, Stephen, Sisic, Zlatko, Johnston, Tracey A.
Published in Journal of inborn errors of metabolism and screening (2016)
Published in Journal of inborn errors of metabolism and screening (2016)
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