Single-Dose Gene-Replacement Therapy for Spinal Muscular Atrophy
Mendell, Jerry R, Al-Zaidy, Samiah, Shell, Richard, Arnold, W. Dave, Rodino-Klapac, Louise R, Prior, Thomas W, Lowes, Linda, Alfano, Lindsay, Berry, Katherine, Church, Kathleen, Kissel, John T, Nagendran, Sukumar, L’Italien, James, Sproule, Douglas M, Wells, Courtney, Cardenas, Jessica A, Heitzer, Marjet D, Kaspar, Allan, Corcoran, Sarah, Braun, Lyndsey, Likhite, Shibi, Miranda, Carlos, Meyer, Kathrin, Foust, K.D, Burghes, Arthur H.M, Kaspar, Brian K
Published in The New England journal of medicine (02.11.2017)
Published in The New England journal of medicine (02.11.2017)
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Journal Article
AVXS-101 (Onasemnogene Abeparvovec) for SMA1: Comparative Study with a Prospective Natural History Cohort
Al-Zaidy, Samiah A, Kolb, Stephen J, Lowes, Linda, Alfano, Lindsay N, Shell, Richard, Church, Kathleen R, Nagendran, Sukumar, Sproule, Douglas M, Feltner, Douglas E, Wells, Courtney, Ogrinc, Francis, Menier, Melissa, L'Italien, James, Arnold, W David, Kissel, John T, Kaspar, Brian K, Mendell, Jerry R
Published in Journal of neuromuscular diseases (2019)
Published in Journal of neuromuscular diseases (2019)
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Journal Article
Single-Dose Gene-Replacement Therapy for Spinal Muscular Atrophy
Mendell, Jerry R., Al-Zaidy, Samiah, Shell, Richard, Arnold, W. Dave, Rodino-Klapac, Louise R., Prior, Thomas W., Lowes, Linda, Alfano, Lindsay, Berry, Katherine, Church, Kathleen, Kissel, John T., Nagendran, Sukumar, L’Italien, James, Sproule, Douglas M., Wells, Courtney, Cardenas, Jessica A., Heitzer, Marjet D., Kaspar, Allan, Corcoran, Sarah, Braun, Lyndsey, Likhite, Shibi, Miranda, Carlos, Meyer, Kathrin, Foust, K.D., Burghes, Arthur H.M., Kaspar, Brian K.
Published in The New England journal of medicine (02.11.2017)
Published in The New England journal of medicine (02.11.2017)
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Journal Article
15.33 AVXS-101 in presymptomatic spinal muscular atrophy (SMA)
Strauss, Kevin A, Swoboda, Kathryn J, Farrar, Michelle A, McMillan, Hugh J, Parsons, Julie, Krueger, Jena M, Iannaccone, Susan T, Chiriboga, Claudia A, Kwon, Jennifer M, Saito, Kayoko, Scoto, Mariacristina, Kausar, Imran, Schultz, Meredith, Kernbauer, Elaine, Farrow, Marcia, Ogrinc, Francis G, Kavanagh, Sarah, Feltner, Douglas E, McGill, Bryan E, Spector, Sidney A, L’Italien, James, Sproule, Douglas M, Muntoni, Francesco
Published in Journal of neurology, neurosurgery and psychiatry (01.12.2019)
Published in Journal of neurology, neurosurgery and psychiatry (01.12.2019)
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Journal Article
250 AVXS-101 phase 3 study in spinal muscular atrophy type 1
Day, John W, Chiriboga, Claudia A, Crawford, Thomas O, Darras, Basil T, Finkel, Richard S, Connolly, Anne M, Iannaccone, Susan T, Kuntz, Nancy L, Peña, Loren DM, Schultz, Meredith, Shieh, Perry B, Smith, Edward C, Kausar, Imran, Feltner, Douglas E, Ogrinc, Francis G, Ouyang, Haojun, Macek, Thomas A, Kernbauer, Elaine, Muehring, Lynlee M, L’Italien, James, Sproule, Douglas M, Kaspar, Brian K, Mendell, Jerry R
Published in Journal of neurology, neurosurgery and psychiatry (01.12.2019)
Published in Journal of neurology, neurosurgery and psychiatry (01.12.2019)
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Journal Article
AVXS-101 Phase 1 gene therapy clinical trial in SMA Type 1: Interim data demonstrates improvements in supportive care use
Shell, Richard, Al-Zaidy, Samiah, Arnold, W. Dave, Rodino-Klapac, Louise, Prior, Thomas W, Lowes, Linda, Alfano, Lindsay, Berry, Katherine, Church, Kathleen, Kissel, John T, Nagendran, Sukumar, L'Italien, James, Sproule, Douglas M, Burghes, Arthur H.M, Foust, K.D, Meyer, Kathrin, Likhite, Shibi, Kaspar, Brian K, Mendell, Jerry R
Published in European journal of paediatric neurology (01.06.2017)
Published in European journal of paediatric neurology (01.06.2017)
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Journal Article
Primary structure of the hydrophobic plant protein crambin
Teeter, Martha M, Mazer, Jonathan A, L'Italien, James J
Published in Biochemistry (Easton) (15.09.1981)
Published in Biochemistry (Easton) (15.09.1981)
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Journal Article
AVXS-101 Phase 1 gene therapy clinical trial in SMA Type 1: Event free survival and achievement of developmental milestones
Mendell, Jerry R, Al-Zaidy, Samiah, Shell, Richard, Arnold, W. David, Rodino-Klapac, Louise, Prior, Thomas W, Lowes, Linda, Alfano, Lindsay, Berry, Katherine, Church, Kathleen, Kissel, John T, Nagendran, Sukumar, L'Italien, James, Sproule, Douglas M, Cardenas, Jessica, Burghes, Arthur H.M, Foust, K.D, Meyer, Kathrin, Likhite, Shibi, Kaspar, Brian K
Published in European journal of paediatric neurology (01.06.2017)
Published in European journal of paediatric neurology (01.06.2017)
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Journal Article
Trypanosoma congolense: structure and molecular organization of the surface glycoproteins of two early bloodstream variants
Strickler, James E, Binder, David A, L'Italien, James J, Shimamoto, Grant T, Wait, Suzanne W, Dalheim, Laura J, Novotny, Jiri, Radding, Jeffrey A, Konigsberg, William H
Published in Biochemistry (Easton) (01.02.1987)
Published in Biochemistry (Easton) (01.02.1987)
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Journal Article
Production and Characterization of Growth Hormone Releasing Factor Analogs Through Recombinant DNA and Chemical Techniques
Kempe, Tomas, Chow, Flora, Peterson, Susan M., Baker, Pamela, Hays, William, Opperman, Gary, L'Italien, James J., Long, Gary, Paulson, Brad
Published in Nature biotechnology (01.06.1986)
Published in Nature biotechnology (01.06.1986)
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Journal Article
014 AVXS-101 gene-replacement therapy (GRT) in presymptomatic spinal muscular atrophy (SMA): study update
Farrar, Michelle, Swoboda, Kathryn J, Schultz, Meredith, McMillan, Hugh, Parsons, Julie, Alexander, Ian E, Kernbauer, Elaine, Farrow, Marcia, Ogrinc, Francis G, Feltner, Douglas E, McGill, Bryan E, Spector, Sidney A, L’Italien, James, Sproule, Douglas M, Strauss, Kevin A
Published in Journal of neurology, neurosurgery and psychiatry (01.07.2019)
Published in Journal of neurology, neurosurgery and psychiatry (01.07.2019)
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Journal Article
066 Avxs-101 gene-replacement therapy (GRT) for spinal muscular atrophy type 1 (SMA1): pivotal phase 3 study (STR1VE) update
Day, John W, Chiriboga, Claudia A, Crawford, Thomas O, Darras, Basil T, Finkel, Richard S, Connolly, Anne M, Iannaccone, Susan T, Kuntz, Nancy L, Pena, Loren DM, Schultz, Meredith, Shieh, Perry B, Smith, Edward C, Farrar, Michelle, Feltner, Douglas E, Ogrinc, Francis G, Macek, Thomas A, Kernbauer, Elaine, Muehring, Lynlee M, L’Italien, James, Sproule, Douglas M, Kaspar, Brian K, Mendell, Jerry R
Published in Journal of neurology, neurosurgery and psychiatry (01.07.2019)
Published in Journal of neurology, neurosurgery and psychiatry (01.07.2019)
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Journal Article
Kinetics of pramlintide degradation in aqueous solution as a function of temperature and pH
Kenley, Richard A., Tracht, Scott, Stepanenko, Anna, Townsend, Michael, L'Italien, James
Published in AAPS PharmSciTech (01.06.2000)
Published in AAPS PharmSciTech (01.06.2000)
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Journal Article
Kinetics of pramlintide degradation in aqueous solution as a function of temperature and pH
Kenley, R A, Tracht, S, Stepanenko, A, Townsend, M, L'Italien, J
Published in AAPS PharmSciTech (01.06.2000)
Published in AAPS PharmSciTech (01.06.2000)
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Journal Article