Duchenne Muscular Dystrophy (DMD)

Companies Expand Development of Exon Skipping for DMD

Two pharmaceutical companies recently announced they are expanding their development and testing of exon-skipping drugs for 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.

Weeping Willows

Chris, a self-taught artist, received an Associate of Arts Degree from Ozarks Technical Community College.  His paintings have been exhibited at several local businesses.  Chris enjoyed reading science fiction and creating artwork.

Woods

James studied design at Sacred Heart University, Fairfield University and the Creative Arts Workshop.  His computer art creations have appeared in the “National Association of Desktop Publishers Journal” and “Computer Graphics World” magazine.  In the summer of 1995 three of his prints, including "Woods," were exhibited at CompUSA on Fifth Avenue in New York.  This work was featured in the 1996 MDA Holiday Wishes card collection.

Untitled - Alex Villareal

Alex created this painting at age 15 at an MDA-sponsored children's art workshop held April 25, 1992, in San Antonio, Texas.  Alex received an art award while in junior high school.

The World in Chaos

Artist: 

Tomás created this artwork at age 10 in an MDA-sponsored workshop held at the Methodist Hospital in Houston.  He served as MDA’s National Goodwill Ambassador in 1995.  Tomás will complete a master’s degree in theology from the University of St. Thomas in Houston in December 2012.  He enjoys literature and writing.

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