Drugs

Sarepta Therapeutics says the U.S. Food and Drug Administration has changed its view of a possible accelerated approval pathway for Duchenne MD drug eteplirsen

posted on November 12, 2013 - 2:46pm
Update (Dec. 10, 2013: Sarepta has posted webcasts of a presentation given Dec. 2, 2013, at a Deutsche Bank BioFEST conference; and of a presentation given Dec.

A nurse and genetic counselor, Trout discusses the diagnostic process and how she helps families with education and support

posted on October 3, 2013 - 9:07am
Quest Vol. 20, No. 4
Nurse Christina Trout Christina Trout is an advanced practice nurse specializing in pediatric neuromuscular disorders and genetic nursing. She's been providing genetic counseling, as well as general education and care coordination, to families with neuromuscular disorders at the University of Iowa since 1993.

Jacobus Pharmaceutical has opened its original, 30-person, invitation-only trial of 3,4-diaminopyridine for Lambert-Eaton myasthenic syndrome (LEMS) to additional participants who meet study criteria

posted on October 1, 2013 - 5:00am
Update (April 14, 2014): This phase 2 trial of 3,4-diaminopyridine is now closed to new participants. However, the drug remains available on a "compassionate use" -- also called "expanded access" -- basis to qualified patients with LEMS and related disorders of the neuromuscular junction. See Compassionate Use of 3,4-Diaminopyridine; or enter NCT01765140 in the search box at ClinicalTrials....

The drug SMT C1100 is still experimental, but an MDA-supported company has received two US patents that provide commercial protection for its development

posted on September 3, 2013 - 5:00am
Summit PLC, a biotechnology company based in Abingdon, United Kingdom, has announced it now has two U.S. patents for its experimental drug SMT C1100 for Duchenne muscular dystrophy (DMD).

In an Aug. 28, 2013, announcement, the Dutch biotechnology company announced favorable results for drisapersen and a new grant for continuing development of PRO045

posted on August 30, 2013 - 5:00am
Update (Oct. 15, 2013):  In an Oct. 15, 2013, webinar, Prosensa announced preliminary results for the phase 1-2 trial of PRO044, designed to target exon 44 of the dystrophin gene. Dystrophin production that is believed to have occurred in response to the drug was seen in 12 out of 21 muscle biopsies that could be evaluated. No drug-related serious adverse events have been reported.

MDA grant will support a study of prednisone in 25 boys with Duchenne muscular dystrophy who are less than 2½ years old

posted on August 20, 2013 - 5:00am
Pediatric neurologist Anne Connolly has received an MDA grant to study the effects of prednisone on Duchenne muscular dystrophy in very young children. MDA has awarded a grant to Anne Connolly, a pediatric neurologist at Washington University in St.

Researchers look at the 'window of opportunity' for effective treatment in spinal muscular atrophy and the therapeutic potential of celecoxib

posted on June 20, 2013 - 4:27pm
Recent spinal muscular atrophy (SMA) research includes findings that shed additional light on the optimal "window of opportunity" for treatment and point toward a potential candidate for therapeutic development.

Results from a 20-person pilot trial suggest that clenbuterol may improve motor and respiratory function in people with spinal-bulbar muscular atrophy, but safety concerns were raised

posted on May 29, 2013 - 4:27pm
A 20-person, open-label pilot trial conducted in Italy and designed to test the safety, tolerability and efficacy of clenbuterol in people with spinal-bulbar muscular atrophy (SBMA, or Kennedy disease) found that the drug was well-tolerated, although some possible safety issues were identified. Trial investigators note that the study findings suggest a possible “positive effect on SBMA disease...