In a Dec. 2, 2014, webcast for the Duchenne MD community, Sarepta Therapeutics explains plans, answers questions; the presentation is available on Sarepta's site

posted on December 2, 2014 - 2:11pm
Cambridge, Mass.-based Sarepta Therapeutics, developer of eteplirsen and other "exon-skipping" drugs designed  to treat Duchenne muscular dystrophy (DMD), provided overall reassurance about eteplirsen's development and some details of future plans to the DMD community in a Dec.

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. Here, MDA answers some frequently asked questions about how the FDA works to shed light on this topic.  Q: Why does the FDA have stringent...

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?

MDA is supporting the inclusion of ‘accelerated approval pathway’ language in the Prescription Drug User Fee Act; community members should contact their legislators

posted on May 24, 2012 - 10:06am
Update (July 10, 2012) — Reauthorization of the Prescription Drug User Fee Act (PDUFA) was signed by President Obama on July 9, 2012, complete with a provision that creates an accelerated approval pathway for drugs for rare diseases.

The pace of research can seem unreasonably slow; here are a few reasons why

posted on January 1, 2012 - 3:11pm
QUEST Vol. 19, No. 1
John Porter from the National Institutes of Health likes to start talks by noting, “It’s a great time to be a mouse with a neuromuscular disease.” Exciting research results are regularly reported, where a treatment appears to cure one neuromuscular disease or another in a mouse — yet there are few treatments available today for people with any of these diseases, and only a few treatments in human...

A decision on approval for Lumizyme, a treatment for Pompe disease, is expected in June

posted on March 19, 2010 - 9:25am
Biotechnology company Genzyme, of Cambridge, Mass., expects the U.S. Food and Drug Administration (FDA) to rule by June 17, 2010, on Genzyme's application to market its laboratory-developed enzyme Lumizyme (alglucosidase alfa) in the United States. Lumizyme and its near-twin, Myozyme, replace the acid maltase enzyme deficient in people with Pompe disease (acid alpha-glucosidase deficiency or acid...

Two federal agencies, the NIH and FDA, are cooperating in an attempt to reduce the time it takes to approve new drugs.

posted on February 26, 2010 - 4:45pm
The National Institutes of Health (NIH) and the U.S. Food and Drug Administration (FDA) have announced plans to establish a Joint NIH-FDA Leadership Council, with the goal of shortening the time it takes to move promising therapies through the regulatory process and into the hands of waiting patients.

A compound being developed to treat Friedreich's ataxia specifically targets an unwanted molecular brake

posted on September 29, 2009 - 11:32am
Scientists at the Scripps Research Institute in La Jolla, Calif., and the Repligen Corporation in Waltham, Mass., have identified the precise biochemical brake that limits production of a needed protein in Friedreich's ataxia (FA) and determined that this brake is specifically targeted by an experim