Drugs

A look at symptoms, diagnosis, treatments and current MDA-supported research into this complex autoimmune disorder

posted on June 15, 2015 - 1:01pm
In recognition of June being National Myasthenia Gravis Awareness Month, MDA is examining the disease from several perspectives. This article offers an overview of signs, symptoms, diagnosis and treatments for myasthenia gravis (MG), as well as information on drugs that people with MG should avoid.

How the FDA tries to balance urgent needs against real risks when evaluating new drugs

posted on April 1, 2013 - 9:25am
Quest Vol. 20, No. 2
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. ===================================================================================== Original...

Data has failed to confirm that treatment with Catena is beneficial in Friedreich's ataxia; sales of the drug in Canada will be discontinued April 30, 2013

posted on March 1, 2013 - 5:00am
Idebenone (under the brand name Catena) in July 2008 received conditional market approval in Canada for the treatment of Friedreich's ataxia (FA). Now, based on additional data that fails to confirm that treatment with Catena is beneficial in FA, Santhera Pharmaceuticals has announced it will discontinue sales of the drug April 30, 2013.

Trial sites have begun opening in a large study to determine which regimen of corticosteroid treatment provides maximum benefit in Duchenne muscular dystrophy

posted on February 15, 2013 - 3:49pm
Update (June 25, 2014): An approximately five-minute video about this FOR-DMD trial has been posted to YouTube.

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

posted on May 3, 2012 - 4:00am
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).

The experimental drug SMT C1100 is designed to increase utrophin production as a treatment for Duchenne MD

posted on April 24, 2012 - 4:00am

A phase 1 clinical trial has begun in Turin, Italy, to test the safety of RG2833 in adults with Friedreich's ataxia

posted on March 15, 2012 - 11:00am
The biopharmaceutical company Repligen Corp., headquartered in Waltham, Mass., has launched a phase 1 clinical trial to test its experimental compound, RG2833, in adults with Friedreich's ataxia (FA). The trial is the first of a therapy specifically developed to treat the underlying molecular cause of FA. The phase 1 trial is being conducted at San Luigi Gonzaga University Hospital in Turin,...

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...