Gene Therapy for Spinal Muscular Atrophy: Safety and Early Outcomes

Historically, autosomal recessive 5q-linked spinal muscular atrophy (SMA) has been the leading inherited cause of infant death. SMA is caused by the absence of the gene, and gene replacement therapy, onasemnogene abeparvovec-xioi, was Food and Drug Administration approved in May 2019. Approval inclu...

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Bibliographic Details
Published in:Pediatrics (Evanston) 2020-09, Vol.146 (3), p.1
Main Authors: Waldrop, Megan A, Karingada, Cassandra, Storey, Mike A, Powers, Brenna, Iammarino, Megan A, Miller, Natalie F, Alfano, Lindsay N, Noritz, Garey, Rossman, Ian, Ginsberg, Matthew, Mosher, Kathryn A, Broomall, Eileen, Goldstein, Jessica, Bass, Nancy, Lowes, Linda P, Tsao, Chang-Yong, Mendell, Jerry R, Connolly, Anne M
Format: Article
Language:English
Subjects:
Adenoviruses, Human
Age
Age Factors
Alanine
Alanine transaminase
Alanine Transaminase - metabolism
Aspartate aminotransferase
Aspartate Aminotransferases - metabolism
Biological Products
Children
Clinical trials
FDA approval
gamma-Glutamyltransferase - metabolism
Gene therapy
Genetic Therapy - adverse effects
Genetic Therapy - methods
Genetic Vectors - administration & dosage
Glucocorticoids - administration & dosage
Humans
Infant
Neuromuscular diseases
Ohio
Outcome Assessment, Health Care
Pediatrics
Prednisolone
Prednisolone - administration & dosage
Recombinant Fusion Proteins - genetics
SMN protein
Spinal Muscular Atrophies of Childhood - genetics
Spinal Muscular Atrophies of Childhood - therapy
Spinal muscular atrophy
Survival of Motor Neuron 1 Protein - genetics
Transaminase
γ-Glutamyltransferase
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Description
Summary:Historically, autosomal recessive 5q-linked spinal muscular atrophy (SMA) has been the leading inherited cause of infant death. SMA is caused by the absence of the gene, and gene replacement therapy, onasemnogene abeparvovec-xioi, was Food and Drug Administration approved in May 2019. Approval included all children with SMA age
ISSN:0031-4005
1098-4275
DOI:10.1542/peds.2020-0729