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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MDA Muscle Walk connects MDA families and raises critical funds for lifesaving treatments and cures. So are you ready to join us?

posted on January 5, 2015 - 9:25am
When Lizzie Chamberlain of Jacksonville, Ill., was 2 years old, her physical development relative to other children began to plateau — and then decline. Lizzie received a diagnosis of type 3 spinal muscular atrophy (SMA). 

Butting in with a view on employment

posted on January 5, 2015 - 9:23am
Considering the title of this column, From Where I Sit, I’m surprised more people have not written about butts. After all, for those of us wheeling around at a seated height — I live with type 2 spinal muscular atrophy (SMA) — other people’s butts are often what we see from where we sit. 

According to experts, reframing your perspective on life can help conquer the anxieties or depression that can accompany living with neuromuscular disease

posted on January 5, 2015 - 9:21am
Even a quick search online will turn up all manner of white papers, analyses and studies that identify a higher risk of anxiety and depression among individuals living with chronic disease and physical disabilities — such as those associated with many types of neuromuscular disease.  

Combating chronic pain in people living with neuromuscular disease requires a multifaceted approach

posted on January 5, 2015 - 9:19am
It is not breaking news to people with neuromuscular disease that combating pain can be a substantial part of daily life. Unfortunately, chronic pain is a complex symptom to manage, and it may be far more pervasive in neuromuscular diseases than originally thought.  “When I started out, about 25 years ago now, there was nothing,” says Gregory Carter, a doctor who specializes in physical...

MDA's Team Momentum made huge strides in its first year and is poised for more success in 2015

posted on January 5, 2015 - 9:17am
Through MDA’s Team Momentum program, participants in endurance races (marathons and half marathons) around the county help raise money to fight muscle disease. These participants receive world-class training, team camaraderie and support for their races, as well as tips and resources for fundraising.  In its inaugural year, 2014, Team Momentum and its dedicated participants enjoyed great...

Expert tips for shedding or adding weight to manage one's disease

posted on January 5, 2015 - 9:15am
Any health professional will tell you that good nutrition is a key to living well with and managing a neuromuscular disease. But what if the disease itself makes that goal hard to reach? That’s the all-too-common challenge many people with neuromuscular disorders face.