research

BMD: Follistatin Gene Transfer Results Encouraging

Results from a trial involving injection of genes for the follistatin protein into the thigh muscles of both legs in six men with Becker muscular dystrophy (BMD) show the experimental treatment appears safe and and was associated with improvement in walking ability (distance walked in six minutes) in four of the six trial participants.

Drug Development for DMD: Fall 2014 Update

Drug development for Duchenne muscular dystrophy (DMD) is progressing on several fronts. Here are some updates as of early November 2014.

PTC is moving forward with ataluren

SMA: Further Data on ISIS-SMNRx Shows Promising Results

Isis Pharmaceuticals, developer of the experimental spinal muscular atrophy (SMA) drug ISIS-SMNRx, revealed positive results from phase 2 clinical studies of the drug focusing on infants and children with SMA. The new data, which was shared on October 10 by ISIS representatives attending the 19th International World Muscle Society Congress in Berlin, included the following highlights:

LEMS: Firdapse Study Shows Encouraging Results

DMD: ReveraGen Drug Will Move to Human Testing

International Consortium Provides $2M Funding for Phase 1 Clinical Trial of ReveraGen DMD Drug

ALS: BrainStorm to Test NurOwn Cells in US

Isis Pharmaceuticals Initiates Phase 1 Study of ISIS-DMPK Rx to Treat Myotonic Dystrophy Type 1

Prosensa Will Pursue Accelerated Approval for Drisapersen to Treat DMD

SMA: ISIS-SMNRx Shows Benefit in Infants, Children

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