Duchenne Muscular Dystrophy (DMD)

CoQ10, Lisinopril Trial in DMD, BMD, LGMD2C-2F, LGMD2I

Researchers at five U.S. and one Canadian center are conducting a clinical trial of the medications coenzyme Q10 and lisinopril to determine their possible beneficial effects on heart function in Duchenne muscular dystrophy (DMD), Becker muscular dystrophy (BMD) and five forms of limb-girdle muscular dystrophy (LGMD).

Experimental Drug Seems to Slow Respiratory Decline in DMD

Update (Sept. 19, 2012):This story was updated to reflect the fact that the phase 3 trial of idebenone in Duchenne MD remains open and that new sites are now participating.

NIAMS Establishes MD Research Center

A Center for Research Translation of Systemic Exon Skipping in Muscular Dystrophy has been established by the National Institute of Arthritis and Musculoskeletal and Skin Diseases (NIAMS), part of the U.S. National Institutes of Health.

PTC Remains Committed to Ataluren for Nonsense-Mutation DMD/BMD

Update (July 25, 2012): This story was updated to reflect the fact that PTC has announced its intention to begin an open-label trial of ataluren in DMD/BMD for former ataluren trial participants in Europe, Israel and Australia.

MDA Awards $13.7 Million in Research Grants

The Muscular Dystrophy Association has awarded 40 research grants totaling $13.7 million to advance the understanding of disease processes and uncover new strategies for treatments and cures of muscular dystrophy and the more than 40 other diseases in the Association's program.

The new grants were recommended by MDA's Scientific and Medical Advisory Committees and approved by MDA's Board of Directors at its July 2011 meeting.

Untitled - Lee Breidenbach

Lee created this artwork at age 10 at an MDA-sponsored children's art workshop held April 25, 1992, in San Antonio, Texas.  In addition to painting, he enjoyed working on the computer and traveling.

Looking a Head

Artist: 

Wilson created this artwork at age 17 at an MDA-sponsored art workshop held July 18, 1996, at the Art Museum of Western Virginia in Roanoke.  He has an associate’s degree in radio/TV production.  His hobbies include playing guitar and writing music.

Four Puppets

Artist: 

These puppets were created in a children's art workshop held in November 1992, in Des Moines, Iowa by four youngsters.  The puppets are mixed media works in papier mache and fabric.

DMD: Phase 2 Exon-Skipping Trial Launched

Updated: View MDA's video about the launch of the eteplirsen trial on YouTube.

Research

MDA-supported investigators are actively pursuing several approaches to halt or reverse the muscle damage caused by Duchenne muscular dystrophy.

Some of the front-running strategies include: inserting new dystrophin genes; changing the way cells interpret genetic instructions; changing the mutated dystrophin gene itself; manipulating other proteins in the body to compensate for the lack of dystrophin; increasing blood flow to skeletal muscles and the heart; combating inflammation; and using stem cells to repair damaged muscles.

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