Nationwide Children's Hospital

Investigators are seeking people with Becker muscular dystrophy to participate in a patient registry and potentially in a gene transfer trial

posted on July 1, 2013 - 5:00am
Update (Nov. 19, 2013):Enrollment for the follistatin gene transfer trial for patients with Becker MD is complete. Enrollment for patients with sporadic inclusion-body myositis, however, is not complete. Investigators remain interested in recruiting participants with either BMD or sIBM for the outcome measures study, also known as the patient registry.

In this podcast from Nationwide Children’s Hospital, pediatric neurologist Francesco Muntoni describes how current understanding of CMD is creating opportunities for therapy development

posted on October 15, 2012 - 5:00am
Editor's note (Oct. 19, 2012): This story was updated to reflect the fact that Kevin Flanigan co-directs the MDA Clinic at Nationwide Children's Hospital in Columbus, Ohio.

Investigators in Columbus, Ohio, need to determine the best way to assess thigh-muscle strength in men with BMD before conducting a gene therapy study

posted on January 18, 2011 - 4:32pm
A new study to determine the best "outcome measure" (measurable activity) with which to assess thigh-muscle (quadriceps) strength in men with Becker muscular dystrophy (BMD) is seeking participants. The study, taking place at Nationwide Children's Hospital in Columbus, Ohio, is a necessary prelude to a planned trial of gene therapy involving injections of genes for the follistatin protein in...

Physicians and scientists will meet Jan. 21-22, 2011, in Columbus, Ohio, at an MDA-sponsored conference about cardiomyopathy in Duchenne muscular dystrophy

posted on January 18, 2011 - 4:06pm
Experts from around the world will gather Jan. 21-22, 2011, at an MDA-sponsored conference about the heart in Duchenne muscular dystrophy (DMD).

The National Institutes of Health has awarded more than $4.5 million to specialized muscular dystrophy research centers at three U.S. institutions

posted on September 30, 2010 - 3:47pm
The U.S. National Institutes of Health (NIH) announced Sept. 29, 2010, that it will allocate more than $4.5 million for the first year of a five-year commitment to explore new treatment strategies for various forms of muscular dystrophy. Support will go to three U.S. institutions: Nationwide Children's Hospital in Columbus, Ohio; the University of Pennsylvania in Philadelphia; and the University...

A study of exon-skipping drug GSK2402968 in nonambulatory subjects with DMD has opened at Nationwide Children's Hospital in Ohio

posted on June 18, 2010 - 11:58am
Update (Nov. 5, 2012): Results announced in November 2012 showed the drug, now called drisapersen, reached blood levels approximately proportional to the injected dose at two of the three dosage levels tested and was not associated with serious adverse events. See Drisapersen Appears Safe in Non-Walking Boys With DMD.

"This Month in Muscular Dystrophy" is the subject of a new audio series from Nationwide Children's Hospital in Columbus, Ohio

posted on May 24, 2010 - 3:53pm
Scientists at the Neuromuscular Disorders Program at Nationwide Children’s Hospital in Columbus, Ohio, are producing a series of podcasts examining current research in neuromuscular disease. The series, “This Month in Muscular Dystrophy,” is an opportunity to hear authors of recent publications discuss how their work improves understanding of these diseases, and what it might mean for treatment.