2021 Abstract Search
Feasibility of measuring functional performance of FSHD patients using wearable sensors to…
91Objective: Evaluate feasibility to monitor daily activity and assess functional outcomes using…
LGMD and Access to Genetic Diagnoses, A Novel Approach.
VirtualAbstract: Limb girdle muscular dystrophies are among a
subset of rare genetic disorders,…
14-gauge core needle biopsy: an effective method for muscle research analysis
86OBJECTIVE
To describe our approach using a 14-gauge disposable core biopsy to collect…
24-Week Results From MT-1186-A01: A Phase 3, Open-Label, Multicenter Safety Study of Oral…
33Background: Radicava® (edaravone injection) is a US FDA-approved treatment for amyotrophic…
A Case of Presenilin 1 (PSEN1) Mutation Associated Spastic Paraparesis
VirtualBackground:
Mutations in the Presenilin 1 (PSEN1) gene are the most common causes of…
A collaborative analysis by clinical trial sponsors and academic experts of anti-transgene…
44Background: Four sponsors have ongoing clinical trials to evaluate the safety and/or efficacy of…
A Novel AAV8-Based Gene Therapy for Duchenne Muscular Dystrophy: Preclinical Studies in the…
VirtualDuchenne muscular dystrophy is an X-linked developmental disorder caused by mutations in the…
A novel approach for investigating the D4Z4 region in facioscapulohumeral muscular dystrophy…
VirtualFacioscapulohumeral muscular dystrophy (FSHD) is caused by aberrant expression of double…
A novel calcium channel gating modifier that improves neuromuscular transmitter release and…
146Background:
Spinal Muscular Atrophy (SMA) is a genetic disease that is caused by a…