DMD: Sarepta Updates Community on Eteplirsen, Other Compounds

DMD: Sarepta Updates Community on Eteplirsen, Other Compounds

Sarepta Therapeutics updated the Duchenne MD community on regulatory and clinical trial progress with respect to eteplirsen, SRP-053 and SRP-045 on Aug. 7, 2014

MG: Multicenter Trial of Rituximab Opens

MG: Multicenter Trial of Rituximab Opens

A phase 2 clinical trial of rituximab in myasthenia gravis has now opened at 15 U.S. centers under the auspices of NIH's NeuroNEXT

DMD: Ataluren Receives Conditional Approval in Europe

DMD: Ataluren Receives Conditional Approval in Europe

Duchenne patients in the European Union with specific dystrophin gene flaws can receive the drug ataluren prior to its full approval

ISIS-SMNRx to Be Tested in Phase 3 Trial in Infants With SMA

ISIS-SMNRx to Be Tested in Phase 3 Trial in Infants With SMA

Isis Pharmaceuticals is testing its experimental, antisense-based drug in a phase 3 trial in SMA-affected babies age 7 months or less

DMD: ReveraGen Drug Will Move to Human Testing

DMD: ReveraGen Drug Will Move to Human Testing

Funding from MDA and three other organizations will allow ReveraGen BioPharma to test its experimental anti-inflammatory drug for Duchenne MD in healthy volunteers

DMD: Eteplirsen Data Still Encouraging at 144 Weeks

DMD: Eteplirsen Data Still Encouraging at 144 Weeks

Sarepta Therapeutics continues to report encouraging data from its extension study of eteplirsen, designed to treat Duchenne MD due to specific genetic mutations

FEATURED IN QUEST MAGAZINE

Balancing parenthood and neuromuscular disease is not just possible — many dedicated moms and dads who live that reality every day say having kids is the best decision they've ever made

posted on July 7, 2014 - 9:23am
Matthew Martin and Amanda Hall have more than a few things in common. They’re roughly the same age — Martin is 28; Hall is 30. They both call the state of Texas home — Martin in Rowlett; Hall in Glenrose. Then there’s the fact that each lives with a neuromuscular disease — Martin was diagnosed with Duchenne muscular dystrophy (DMD) at the age of 8; Hall with congenital myopathy in her early 20s.

Facing a neuromuscular disease diagnosis can be devastating, overwhelming and confusing, but empowering yourself to take charge of your care or your child's care is more important than ever

posted on July 7, 2014 - 9:21am
Whether you are newly diagnosed or have been living with a neuromuscular disease for years, there is arguably one guiding principle that is most important when it comes to making decisions about your care and quality of life: You are in charge. And with good reason. 

Questions and answers about the FDA and its process for approving drugs

posted on July 7, 2014 - 9:19am
To people faced with life-threatening diseases, the U.S. Food and Drug Administration (FDA) can seem like an indifferent obstacle, keeping them from treatments that would otherwise be available. But the reality is much more layered and complex.

Five tips for dealing with the diagnosis of a late-onset muscle disease

posted on July 7, 2014 - 9:17am
Each form of muscular dystrophy is devastating in its own way. For some patients, symptoms may manifest at birth or at a young age. My situation was different.

Tips on how to find and use an accessible hotel pool lift this summer

posted on July 7, 2014 - 9:15am
ADA-compliant pool chair lifts provide individuals with disabilities a safe way to enter and exit public pools. Many vacationers are seeing something different at hotels this summer: pool and spa lifts.

Meet clinical psychologist and disability advocate Danielle Sheypuk

posted on July 7, 2014 - 9:12am
Danielle Sheypuk, Ph.D., doesn’t just talk about the idea that people with disabilities can have robust and fulfilling romantic lives — she lives it, too.