October 17, 2014Molecular TherapyOpen Access

A Phase 1/2a Follistatin Gene Therapy Trial for Becker Muscular Dystrophy

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Why the study?

Does AAV1.CMV.FS344 gene therapy improve walking distance in patients with Becker muscular dystrophy?

Population

6 patients with Becker muscular dystrophy (BMD)

Design

Case_series

Authors

JMJerry R. MendellUniversity of Iowa ZSZarife SahenkNationwide Children's Hospital VMVinod MalikNationwide Children's Hospital

Discussion

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Implication

Hypothesis-generating for AAV-follistatin therapy in Becker MD; larger controlled trials required before any clinical consideration.

Structured PICO

Does AAV1.CMV.FS344 gene therapy improve walking distance in patients with Becker muscular dystrophy?

Population

6 patients with Becker muscular dystrophy (BMD)

Intervention

AAV1.CMV.FS344 (follistatin gene therapy) delivered by direct bilateral intramuscular quadriceps injections (Cohort 1: 3 × 10^11 vg/kg/leg; Cohort 2: 6 × 10^11 vg/kg/leg)

Outcome

Distance walked on the 6-minute walk test (6MWT)surrogate

Follistatin gene therapy via AAV delivery appears safe and may improve functional capacity and muscle histology in patients with Becker muscular dystrophy.

Cite This Study

Mendell et al. (2014) studied this question.

synapsesocial.com/papers/6a757d12ef3d015f2ddd74a5 https://doi.org/10.1038/mt.2014.200

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A Phase 1/2a Follistatin Gene Therapy Trial for Becker Muscular Dystrophy

Discussion

Member takes

Implication

Structured PICO

Cite This Study

A Phase 1/2a Follistatin Gene Therapy Trial for Becker Muscular Dystrophy

Discussion

Member takes

Implication

Structured PICO

Cite This Study

Also Consider

Also Consider