SMA: Further data on ISIS-SMNRx shows promising results

SMA: Further data on ISIS-SMNRx shows promising results

Isis Pharmaceuticals updated the SMA community on phase 2 clinical trial results of the drug ISIS-SMNRx.

Phase 3 Clinical Trial for Eteplirsen Now Enrolling Boys with Duchenne Muscular Dystrophy

Phase 3 Clinical Trial for Eteplirsen Now Enrolling Boys with Duchenne Muscular Dystrophy

Boys between the ages of seven and 16 with Duchenne muscular dystrophy are being recruited to participate in a phase 3 clinical trial to evaluate the effectiveness and safety of eteplirsen in treating DMD.

LEMS: Firdapse Study Shows Encouraging Results

LEMS: Firdapse Study Shows Encouraging Results

Catalyst Pharmaceuticals launches Firdapse expanded access program for eligible people living with congenital myasthenic syndromes and Lambert-Eaton myasthenic syndrome

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

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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.