Clinical Trials and Studies

Reducing dynamin 2 protein levels benefited mice with a disorder mimicking human myobutular myopathy; the strategy could have implications for MTM and additional diseases

posted on April 15, 2014 - 10:04am
A French research team has found that reducing levels of a protein called dynamin 2 has potential as a strategy to treat myotubular myopathy (MTM), a form of centronuclear myopathy (CNM), and that it could have implications for other nerve and muscle disorders as well.

How MDA-supported research to counteract a complement protein and rev up regulatory T cells may improve MG treatment

posted on April 1, 2014 - 9:27am
Quest Spring 2014
"I’m on CellCept, prednisone, Mestinon and IVIG every three weeks," says 38-year-old Rachel Pegram. "Prednisone, which I have taken for more than 22 years now, has very rough side effects. It has caused weight gain, diabetes and glaucoma. I cannot say I have ever gone into remission without drugs, but I believe I have been in a drug-induced remission. I have spent a lot of time in hospitals for...
posted on April 1, 2014 - 9:03am
Quest Spring 2014
The research news featured in this section is a compilation of Quest News Online and MDA/ALS Newsmagazine Online articles posted between issues of the print magazine (Nov. 14, 2013-Feb. 10, 2014). Download the Spring 2014 PDF to see Progress Now/Research Updates as it appears in the April-May-June 2014 print issue.

A phase 2 trial of Duchenne MD drug drisapersen found the high-dose group walked farther than the placebo group at 24 and 48 weeks

posted on March 18, 2014 - 4:10pm
Update March 27, 2014: Prosensa and United Parent Projects Muscular Dystrophy jointly presented an educational webinar for patients and families affected by Duchenne muscular dystrophy on March 25, 2014. The approximately one-hour presentation is archived at Events & Presentations on the Prosensa site and includes a thorough discussion of Prosensa's plans for drisapersen and other exon-...

A study to determine the usual disease course of myotubular myopathy has sites in North America and France

posted on March 18, 2014 - 11:41am
A study to track the natural history and functional capabilities of patients with myotubular myopathy (MTM), a form of centronuclear myopathy (CNM), is recruiting approximately 60 patients of any age (including newborns). There are three North American study sites — Boston, Bethesda, Md., Toronto — and a site in Paris.

Preliminary results for a phase 2 trial of the experimental compound olesoxime suggest it may preserve motor function in spinal muscular atrophy

posted on March 13, 2014 - 9:41am
The experimental compound olesoxime (TRO19622), in development by French biotechnology company Trophos as a potential treatment for spinal muscular atrophy (SMA), has shown encouraging results in a phase 2 trial involving 165 children and young adults with this disorder.

Preliminary results from four infants in a phase 2 trial of ISIS-SMNRx suggest the drug is well-tolerated and may prolong ventilator-free survival

posted on March 3, 2014 - 2:17pm
Interim results of a phase 2 clinical trial to test multiple doses of the experimental drug ISIS-SMNRx in infants with spinal muscular atrophy (SMA) suggest that the drug is well-tolerated and may prolong ventilator-free survival.

An MDA-supported team has developed mice with transplanted muscle tissue from people with facioscapulohumeral MD that is likely to speed research  

posted on February 14, 2014 - 1:44pm
Researchers funded in part by MDA have created a new research mouse (mouse "model") that they say is likely to speed investigations in facioscapulohumeral muscular dystrophy (FSHD) and therapy development for this disease. It's expected to complement an existing FSHD mouse model.