Drugs

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

posted on April 1, 2013 - 9:25am
The approval of new drugs in the United States is a complex process that seeks to balance the need to protect the health and safety of the population with the need to provide treatment as soon as possible for serious or life-threatening disorders.

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
Prednisone and other drugs in the corticosteroid family have become standard treatment for Duchenne muscular dystrophy (DMD) in much of the world for almost a decade. In this disorder, they slow the decline of muscle function and prolong walking ability. However, corticosteroids have significant side effects, such as weight gain, bone loss, cataracts and behavior changes.

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...

Yearlong, MDA-supported study comparing two prednisone dosing schedules in Duchenne MD found similar benefits and side effects

posted on July 20, 2011 - 9:52am
A one-year, MDA-supported study comparing a weekend-only prednisone treatment schedule with a daily prednisone schedule in boys with Duchenne muscular dystrophy (DMD) has found that the two treatment regimens provide about the same benefits and have approximately the same side-effect profile.
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