Study of Intrathecal Administration of Onasemnogene Abeparvovec-xioi for Spinal Muscular Atrophy (STRONG)
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|ClinicalTrials.gov Identifier: NCT03381729|
Recruitment Status : Suspended (FDA placed AVXS-101 IT administration studies on clinical hold pending further discussions regarding pre-clinical findings.)
First Posted : December 22, 2017
Last Update Posted : June 9, 2021
Novartis Gene Therapies
Information provided by (Responsible Party):
Novartis ( Novartis Gene Therapies )
No Study Results Posted on ClinicalTrials.gov for this Study
|Recruitment Status :||Suspended|
|Estimated Primary Completion Date :||April 19, 2024|
|Estimated Study Completion Date :||April 19, 2024|
Duque SI, Arnold WD, Odermatt P, Li X, Porensky PN, Schmelzer L, Meyer K, Kolb SJ, Schümperli D, Kaspar BK, Burghes AH. A large animal model of spinal muscular atrophy and correction of phenotype. Ann Neurol. 2015 Mar;77(3):399-414. doi: 10.1002/ana.24332. Epub 2015 Feb 9.
Foust KD, Wang X, McGovern VL, Braun L, Bevan AK, Haidet AM, Le TT, Morales PR, Rich MM, Burghes AH, Kaspar BK. Rescue of the spinal muscular atrophy phenotype in a mouse model by early postnatal delivery of SMN. Nat Biotechnol. 2010 Mar;28(3):271-4. doi: 10.1038/nbt.1610. Epub 2010 Feb 28.
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