FDA Approves Emflaza for Treatment of Duchenne Muscular Dystrophy
The U.S. Food and Drug Administration has granted approval for deflazacort (brand name Emflaza), under development by Marathon Pharmaceuticals, to treat DMD
Category: Research
FSHD Study Seeks Participants
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.
Spinraza a Breakthrough for SMA
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.
Myotonic Dystrophy Clinical Research Network Set to Continue Specialized Work with Nearly $1 Million in New MDA Funding
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.
New Antisense Approach Holds Promise for Treatment of SMA
Encouraging results from an MDA-supported study, have revealed a potential new strategy to treat spinal muscular atrophy (SMA).
C9ORF72 ALS Study Seeks Participants
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 Reports Setback on DMPKRx Program for Myotonic Dystrophy
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.
Alexion Seeks Approval of Soliris as a Treatment for MG
Alexion Pharmaceuticals announced it has submitted a marketing application to the FDA for the investigational drug eculizumab (brand name Soliris).
CMS Study Seeks Participants
Researchers are looking for people with congenital myasthenic syndrome (CMS) to participate in a phase 3 study being conducted by Catalyst Pharmaceuticals to test the experimental drug amifampridine phosphate.
Amifampridine phosphate is a potassium channel inhibitor designed to cause greater stimulation of muscle by prolonging nerve signals and is expected to help treat muscle weakness in people with CMS. The drug has shown promising results in treating patients with a related disorder called LEMS.
Encouraging Clinical Study Data Reported for ALS Drug Edaravone
Osaka-based Mitsubishi Tanabe Pharma has reported encouraging 12-month efficacy and safety data for edaravone (brand name Radicava) for the treatment of ALS. Edaravone is delivered by intravenous injection. It’s thought to work by relieving the effects of oxidative stress, which, in people with ALS, has been suspected to play a role in the death of . . .
