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.
Udai Pandey was awarded an MDA research grant totaling $300,000 over three years to identify new drugs for ALS caused by a mutation in the FUS gene.
Cytokinetics has reported negative results from its international phase 3 VITALITY-ALS trial to test the investigational drug tirasemtiv in people with ALS.
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.
Researchers are looking for people with MNDs like ALS to join a clinical trial for the investigational drug FLX-787-ODT.
Researchers are looking for people with ALS to participate in a phase 2 clinical trial for the investigational drug CK-2127107.
Researchers are looking for individuals with type 1 SMA for a clinical trial to test the investigational drug RG7916.
Researchers are looking for individuals with type 1 SMA to test efficacy for its SMA gene replacement therapy AVXS-101.
MDA has awarded a grant to Iron Horse Diagnostics to support development of a prognostic (predictive) test for ALS.
PTC Therapeutics President and CEO Stuart Peltz, Ph.D., issued a letter to the DMD community after the FDA declined to approve ataluren (brand name Translarna) for the treatment of DMD.
