Clinical Trials and Studies

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

posted on October 17, 2014 - 8:00am
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:

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.

posted on October 7, 2014 - 8:20am
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.

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

posted on October 3, 2014 - 2:00pm
Catalyst Pharmaceuticals, a biopharmaceutical company focused on rare debilitating diseases, announced encouraging results on Sept. 29, 2014, from its phase 3 clinical trial of Firdapse (amifampridine phosphate tablets equivalent to 10mg amifampridine) for the symptomatic treatment of Lambert-Eaton myasthenic syndrome (LEMS).

Funding from MDA and three other organizations will allow ReveraGen BioPharma to test its experimental anti-inflammatory drug for Duchenne MD in healthy volunteers

posted on July 17, 2014 - 8:00am
ReveraGen BioPharma, based in Silver Spring, Md., is moving ahead with a phase 1 trial of an experimental compound in development to treat Duchenne muscular dystrophy (DMD), made possible by a $2 million grant from U.S.-based MDA and three United Kingdom-based DMD organizations.

Sarepta Therapeutics continues to report encouraging data from its extension study of eteplirsen, designed to treat Duchenne MD due to specific genetic mutations

posted on July 10, 2014 - 1:59pm
Walking ability and respiratory function in boys with Duchenne muscular dystrophy (DMD) show continued benefit from eteplirsen at 144 weeks (almost three years), the drug's developer announced today. In addition, the intravenously infused drug was well tolerated, with no serious treatment-related adverse events seen.

Questions and answers about the FDA and its process for approving drugs

posted on July 7, 2014 - 9:19am
Quest Summer 2014
To people faced with life-threatening diseases, the U.S. Food and Drug Administration (FDA) can seem like an indifferent obstacle, keeping them from treatments that would otherwise be available. But the reality is much more layered and complex.

Four professionals recently talked with Margaret Wahl, MDA’s medical and science editor, sharing their thoughts about access to drugs that have not gone through the full approval process

posted on July 7, 2014 - 9:09am
Quest Summer 2014
Can We Have It Now?

Drug development consultant Abby Bronson says early access to a new drug can jeopardize its full approval and long-term availability

posted on July 7, 2014 - 9:09am
Quest Summer 2014
Abby Bronson is the Duchenne muscular dystrophy program manager at Children's National Medical Center in Washington, D.C., where she works with various stakeholders to further therapeutic development for this disease. Bronson has a master's degree in business administration from the University of Pennsylvania and has managed new product development and marketing at biotechnology and...