Background: For people with muscular dystrophy, ALS, locked-in syndrome, and other neurologic conditions causing quadriparesis and severe dysarthria/anarthria, the loss of fluent communication is highly […]
Objective: To assess safety, pharmacokinetics (PK), pharmacodynamics and biomarkers of muscle damage in healthy adults and adults with Becker muscular dystrophy (BMD). Background: Fast (Type […]
Objective: The primary objective of the MARINA study is to evaluate the safety and tolerability of single and multiple ascending doses of AOC 1001 in […]
Duchenne muscular dystrophy (DMD) is a genetic, fatal, muscle wasting neuromuscular disease that affects 1 in 3,500-5000 boys around the world. Despite recent progress, the […]
Background: Four sponsors have ongoing clinical trials to evaluate the safety and/or efficacy of investigational gene therapies for the treatment of Duchenne muscular dystrophy (DMD). […]
Background: Peptide-conjugated phosphorodiamidate morpholino oligomers (PPMOs) are a next-generation chemistry platform in which a cell-penetrating peptide is conjugated to the PMO backbone, with the goal […]
Duchenne muscular dystrophy (DMD) is a progressive, fatal neuromuscular disease caused by the absence of functional dystrophin protein in skeletal and cardiac muscle. SGT-001 is […]
Background: Adeno-associated virus (AAV) vectors have become the vehicle of choice for gene transfer therapy for Duchenne muscular dystrophy (DMD). The rAAVrh74 serotype, which efficiently […]
Background Duchenne muscular dystrophy is an X-linked recessive neuromuscular disorder caused by loss of dystrophin in muscle tissues. Vamorolone is a dissociative steroidal anti-inflammatory drug […]