How the FDA tries to balance urgent needs against real risks when evaluating new drugs
Update (April 15, 2014): This story has been updated to reflect that there is now a new FDA office, the Office of Health and Constituent Affairs, whose job it is to serve as a liaison between the FDA and various outside stakeholders, including patient advocates and consumers. See Resources, below.
Drug testing for Duchenne MD, Leigh syndrome and Pompe disease moves forward; and two MDA-supported mouse studies suggest leads for myasthenia gravis and periodic paralysis
Drug development and identifying new leads for possible drug development are in the news for five neuromuscular diseases in MDA’s program.
MDA has awarded $1.5 million to ReveraGen BioPharma to develop a drug with the benefits of prednisone, but without the side effects, for Duchenne MD
The Muscular Dystrophy Association has awarded $1,549,725 to ReveraGen BioPharma, a Rockville, Md., biotechnology company, for development of a dissociative glucocorticoid to treat Duchenne muscular dystrophy (DMD).
In this second report on MDA's Clinical Conference, updates on experimental therapies in development for six neuromuscular diseases are described
The progress of several experimental therapies currently in development for neuromuscular diseases was discussed at MDA's 2012 Clinical Conference, held in Las Vegas March 4-7.
The pace of research can seem unreasonably slow; here are a few reasons why
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...
AVI BioPharma is developing two new exon-skipping drugs; GlaxoSmithKline will conduct more trials of GSK2402968, for which participants are being recruited
Two pharmaceutical companies recently announced they are expanding their development and testing of exon-skipping drugs for Duchenne muscular dystrophy (DMD).
After restructuring its agreement with Genzyme, PTC will continue developing ataluren for nonsense-mutation DMD/BMD and will study the drug in former trial participants
Update (July 25, 2012): This story was updated to reflect the fact that PTC has announced its intention to begin an open-label trial of ataluren in DMD/BMD for former ataluren trial participants in Europe, Israel and Australia.