MDA has awarded an MDA Venture Philanthropy (MVP) grant to Izumi Biosciences, Inc., to fund early-stage development of IZ10023, for use in people with ALS who are taking riluzole, the only drug approved by the U.S. Food and Drug Administration to treat ALS.
PTC Therapeutics announced that the FDA will review its investigational drug ataluren (brand name Translarna) for the treatment of some forms of DMD.
Researchers are looking for people with Lambert-Eaton myasthenic syndrome (LEMS) to participate in a phase 3 clinical trial, being conducted by Catalyst Pharmaceuticals, to test the investigational drug amifampridine phosphate. The Firdapse Strength Trial for LEMS, or FirST-4-LEMS, study is designed to evaluate the effectiveness of amifampridine phosphate in controlling, reducing and/or eliminating symptoms of . . .
The FDA has approved Marathon Pharmaceuticals’ new drug applications for deflazacort (brand name Emflaza) to treat Duchenne Muscular Dystrophy
Researchers are looking for people with facioscapulohumeral muscular dystrophy (FSHD) to participate in a phase 2 clinical trial, being conducted by Acceleron Pharma, to test the investigational drug ACE-083.
On Dec. 23, 2016 the FDA approved Spinraza for the treatment of SMA. In clinical trials to test Spinraza, participants who received treatment with the drug experienced life-changing outcomes they wouldn’t have been expected to achieve.
The Muscular Dystrophy Association is pleased to announce the award of a clinical research network grant totaling $918,000 over three years to spur advances in myotonic dystrophy (DM) research.
Encouraging results from an MDA-supported study, have revealed a potential new strategy to treat spinal muscular atrophy (SMA).
Researchers are looking for people with ALS to participate in a research study aimed at better understanding the specific form of ALS caused by a mutation in the gene for C9ORF72.
Ionis Pharmaceuticals provided an update on its recently-concluded phase 1-2 clinical trial of IONIS-DMPK-2.5Rx, an experimental compound for the treatment of type 1 myotonic dystrophy (DM1). Analysis showed mixed results from the trial.
