Adeno-associated virus serotype 9 antibodies in patients screened for treatment with onasemnogene abeparvovec.
Adeno-associated virus serotype 9
antibody titers
clinical trials
enzyme-linked immunosorbent assay
gene replacement therapy
gene therapy
managed access programs
onasemnogene abeparvovec
spinal muscular atrophy
survival motor neuron
Journal
Molecular therapy. Methods & clinical development
ISSN: 2329-0501
Titre abrégé: Mol Ther Methods Clin Dev
Pays: United States
ID NLM: 101624857
Informations de publication
Date de publication:
11 Jun 2021
11 Jun 2021
Historique:
received:
10
12
2020
accepted:
19
02
2021
entrez:
26
3
2021
pubmed:
27
3
2021
medline:
27
3
2021
Statut:
epublish
Résumé
Spinal muscular atrophy is a progressive, recessively inherited monogenic neurologic disease, the genetic root cause of which is the absence of a functional
Identifiants
pubmed: 33768131
doi: 10.1016/j.omtm.2021.02.014
pii: S2329-0501(21)00029-2
pmc: PMC7973120
doi:
Types de publication
Journal Article
Langues
eng
Pagination
76-82Informations de copyright
© 2021 The Authors.
Déclaration de conflit d'intérêts
J.W.D. reports financial relationships with Novartis Gene Therapies, Inc., Affinia Therapeutics, Biogen, Cytokinetics, Ionis, Pfizer, Roche, and Sarepta, and royalties from Athena Diagnostics. R.S.F. has served as an advisor to Novartis Gene Therapies, Inc., Ionis, Biogen, and Roche, and received licensing fees from the Children’s Hospital of Philadelphia related to development of the Children’s Hospital of Philadelphia Infant Test of Neuromuscular Disorders scale. E.M. has received personal compensation for clinical trial consulting and serving on scientific advisory boards from Novartis Gene Therapies, Inc. K.J.S. has received personal compensation from Novartis Gene Therapies, Inc., for serving as an advisory board member, and from Biogen for serving as a visiting professor; and has received research support from Novartis Gene Therapies, Inc., and Biogen-sponsored clinical trials. M.M. is an employee of Novartis Gene Therapies, Inc. R.v.O. and S.T.-W. are employees of Novartis Gene Therapies, Inc., and own Novartis stock or other equities. J.R.M. declares no competing interests.
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