Muscular Dystrophies

‘Focused, Intense’ MDA Conference Advances Neuromuscular Disease Research

Turning neuromuscular disease research into treatments as quickly and effectively as possible was the overarching theme of dozens of formal presentations, nearly 200 scientific posters, and countless informal conversations at the MDA Scientific Conference, April 21-24.

A palpable sense of excitement pervaded the sold-out event thanks to the unprecedented number of experimental treatments in clinical trials for neuromuscular diseases, and the unique opportunity the conference provided for information-sharing and collaboration among scientific professionals from many disciplines.

MDA Scientific Conference To Emphasize Therapy Development

The Muscular Dystrophy Association’s annual conference being held in Washington, D.C., on April 21-24, 2013, is centered on the theme Therapy Development for Neuromuscular Diseases: Translating Hope into Promise.

DMD/BMD: MDA Funds Development of Drug to Fix Calcium Leaks

MDA has awarded $1 million to biopharmaceutical company ARMGO Pharma for development of a new strategy for treating Duchenne muscular dystrophy (DMD) or Becker muscular dystrophy (BMD).

DMD: FDA Asks Sarepta for More Data on Eteplirsen

The U.S. Food and Drug Administration (FDA) has said it will consider an application for accelerated approval for eteplirsen, an experimental drug for Duchenne muscular dystrophy (DMD), after it reviews additional data from clinical trials of the drug.

DMD: Drisapersen Outperforms Placebo in Walking Test

The exon-skipping drug drisapersen has shown encouraging results in an international phase 2b trial in boys with Duchenne muscular dystrophy (DMD) caused by specific mutations.

DMD: Eteplirsen Shows Continued Benefit at 74 Weeks; Data To Be Announced at MDA Conference

Eteplirsen, an experimental exon-skipping therapy designed to treat Duchenne muscular dystrophy (DMD) caused by specific mutations in the dystrophin gene, continues to show sustained benefit on walking distance through week 74 of a phase 2b, 12-person study.

Newly Developed FSHD Mice Likely To Aid Research

A newly developed research mouse that has the same combination of genetic alterations that causes human facioscapulohumeral muscular dystrophy (FSHD) is expected to change the way research in this disease is conducted, possibly speeding the development of therapies.

Unlike humans, mice normally do not have a DNA structure called a D4Z4 repeat array that, when altered, causes FSHD. Therefore, FSHD research mice have been particularly difficult to create.

Research Briefs: DMD, Leigh Syndrome, MG, Periodic Paralysis, Pompe Disease

Drug development and identifying new leads for possible drug development are in the news for five neuromuscular diseases in MDA’s program.

Government Funding for NMD Research Largely Spared in Continuing Resolution

The news is generally favorable for many important government-funded neuromuscular disease research projects, in the wake of Congress’ recent passage of a continuing resolution funding the government through the end of September. 

Most government operations, except those funded through the full-year appropriations bills, will be maintained at essentially the same level as fiscal year (FY) 2012. (The government's fiscal year begins on Oct. 1 and ends on Sept. 30 of the following year.)

DMD, BMD: Combining Gene Therapy and Stem Cell Transplantation

A therapeutic strategy that combines gene therapy and stem cell transplantation has shown encouraging results in mice with a disorder mimicking Duchenne muscular dystrophy (DMD).

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