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

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posted on July 7, 2014 - 9:08am
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Today's power wheelchair innovations put users in the driver's seat like never before

posted on July 7, 2014 - 9:07am
Lateral Tilt System from Motion Concepts Dynamic Controls’ iPortal Mouse Mover + Accessibility Aquila Corp.’s Airpulse PK2 wheelc

Feedback from Quest readers and the MDA community

posted on July 7, 2014 - 9:05am
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Research and Clinical Trial Updates

posted on July 7, 2014 - 9:03am
What About My Disease? Readers sometimes wonder what’s happening with research for their diseases when they don’t see news about them for a while in the pages of Quest. But keep in mind: Research that seems to be for one disorder often has spillover implications for others.

From MDA camp to promising drug trials, summer brings reasons to be hopeful

posted on July 7, 2014 - 9:01am
Can you hear it? It’s the sound of children’s laughter at nearly 80 MDA summer camps across the country. At MDA camp, thousands of kids with muscle disease discover a world where barriers don’t exist, and they meet many other kids sharing the same needs and experiences. It’s a place where kids can just be kids, as they build lifelong friendships and gain increased self-confidence.