research

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

MD-CARE Act Congressional Briefing Accelerates Reauthorization Momentum

"The energy in the room was high, and the mood was optimistic," said Annie Kennedy, MDA's senior vice president of advocacy, speaking about the Congressional briefing on reauthorization of the MD-CARE Act that she attended and helped to organize. The briefing was co-hosted by MDA and other muscular dystrophy organizations in Washington, D.C., on Feb.

High-Calorie Diet May Help in ALS

A study supported in part by MDA has found that a high-calorie, high-carbohydrate diet is safe and well-tolerated in late-stage amyotrophic lateral sclerosis (ALS), that it leads to moderate weight gain, and that it may slow disease progression.

SMA: Interim Results Look Promising for SMN-Raising Drug

Interim results of a phase 2 clinical trial to test multiple doses of the experimental drug ISIS-SMNRx in infants with spinal muscular atrophy (SMA) suggest that the drug is well-tolerated and may prolong ventilator-free survival.

DMD: Prosensa to Present Webcasts on Drug Development

Dutch biopharmaceutical company Prosensa, developer of the experimental drug drisapersen and other experimental compounds for Duchenne muscular dystrophy (DMD), will update the investor community via a Thursday, Jan. 16, 2014, presentation at the J.P. Morgan Healthcare Conference in San Francisco. The presentation will be webcast at 10 a.m. Pacific time/1 p.m. Eastern time.

On Tuesday, Jan. 21, at 8 a.m. Eastern time, Prosensa will conduct a separate webinar for DMD patients and families.

DMD: Eteplirsen-Treated Boys Show Continued Stability at 120 Weeks

Boys with Duchenne muscular dystrophy (DMD) participating in a phase 2b extension trial of the experimental exon-skipping drug eteplirsen showed continued stabilization of walking distance at 120 weeks. Sarepta Therapeutics, developer of eteplirsen, announced the findings in a Jan. 15, 2014, press release.

DMD: GSK Returns Drisapersen Development Rights to Prosensa

The rights to development of the experimental Duchenne muscular dystrophy (DMD) drisapersen have now been returned to the drug's original developer, Dutch biotechnology company Prosensa. The rights were formerly jointly held by Prosensa and multinational pharmaceutical company GlaxoSmithKline (GSK).

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