Solid Biosciences announced the launch of its first clinical trial for SGT-001. The phase 1/2 study, called IGNITE DMD, is designed to assess safety and efficacy of a single dose of SGT-001 in children and adolescents with DMD.
Catalyst Pharmaceuticals has reported encouraging results from its second phase 3 clinical trial to test the investigational drug amifampridine phosphate (brand name Firdapse) in people with LEMS.
Cytokinetics has reported negative results from its international phase 3 VITALITY-ALS trial to test the investigational drug tirasemtiv in people with ALS.
The investigational drug JOTROL under development by Jupiter Orphan Therapeutics to treat FA has received FDA Orphan Drug Designation.
Did you miss MDA’s recent Caregiver Webinar in partnership with SYNERGY HomeCare? Find the webinar and highlights here:
The Muscular Dystrophy Association launched a new event series in 2017, MDA Discovery & Impact Events, to bring greater awareness to and educate families and supporters about the progress being made in the neuromuscular disease space.
MDA awarded 13 grants worth $3.5 M to researchers working to find treatments and cures for NMDs worldwide.
Researchers are looking for individuals with type 1 SMA to test efficacy for its SMA gene replacement therapy AVXS-101.
Join us Thursday Nov. 9 for a webinar in partnership with SYNERGY HomeCare geared toward family caregivers.
Congress has voted to extend the FAA’s tax authority for six months. As anticipated, the extension did not include any of the disability provisions set out in the full bill, but the possibility of these provisions being included in the full reauthorization in March remains.
