clinical trial

Sarepta Reaffirms Commitment to Eteplirsen, Other DMD Drugs

ALS: Mexiletine Helped Muscle Cramps But Did Not Show Other Benefits

Phase 3 Trial of ISIS-SMNRx Now Open for Children with SMA

PTC To Test RG7800 in SMA

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.

BioBlast Is Developing an OPMD Drug

AveXis SMA Gene Therapy Trial Continues Recruiting

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

Sarepta Announces Revised Timeline for Eteplirsen FDA Application

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

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:

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