DMD: Eteplirsen Data Still Encouraging After Three Years

DMD: Eteplirsen Data Still Encouraging After Three Years

Boys with DMD continuously treated with eteplirsen for three years walked farther in six minutes than those receiving a placebo for the first six months

Stanford Biobank Seeks Tissue Samples

Stanford Biobank Seeks Tissue Samples

Stanford University is collecting samples of blood, muscle and other tissues from people with neuromuscular disorders for use in research

PTC Begins Submission of New Drug Application for DMD Stop Codon Read-Through Drug

PTC Begins Submission of New Drug Application for DMD Stop Codon Read-Through Drug

PTC Therapeutics has begun submitting an application for U.S. approval of Translarna (ataluren) for Duchenne MD; completion of the application is slated for late 2015

Trial of Antisense Drug for Type 1 Myotonic Dystrophy Opens

Trial of Antisense Drug for Type 1 Myotonic Dystrophy Opens

Isis Pharmaceuticals has announced the opening of a U.S. trial to evaluate the safety and tolerability of ISIS-DMPKRx in adults with type 1 myotonic dystrophy

Myostatin Blocker To Be Tested in DMD

Myostatin Blocker To Be Tested in DMD

Pfizer will test its experimental drug PF-06252616 in ambulatory boys with Duchenne MD at multiple sites in the U.S. and elsewhere

DMD: Utrophin Modulator SMT C1100 To Undergo Further Testing

DMD: Utrophin Modulator SMT C1100 To Undergo Further Testing

MDA-supported Summit PLC announced it will continue testing it experimental drug in Duchenne MD patients, along with dietary modifications that may improve drug absorption

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Accelerating progress in 2015 — and beyond

posted on January 5, 2015 - 9:01am
MDA’s resolve and action steps to accelerate progress on behalf of our families fighting muscle disease has never been greater. People fighting these difficult diseases are depending on MDA to fulfill our mission to save and improve their lives.  To most effectively help our families, MDA must be very intentional about building on the scientific and medical progress we have led for the past...