Duchenne Muscular Dystrophy (DMD)

MDA Funds Development of Utrophin 'Magnet' for DMD/BMD

 

Nationwide Children's Podcast Probes Exon Skipping in DMD

A January 2012 podcast from Nationwide Children's Hospital in Columbus, Ohio, explores the potential of a strategy called exon skipping as a treatment for Duchenne muscular dystrophy (DMD).

2011 Lexus GX-460 Premium

Artist: 

Dhruv began drawing pictures as a very young boy.  He especially enjoys drawing cars and Star Wars characters and playing robotics and video games.  His favorite school subjects are math and science.  This drawing was reversed from black on white to white on black.

MDA Funds Clinical Studies of Reformulated Drug for DMD

MDA has awarded $750,000 to Summit Corporation PLC for development and testing of SMT C1100, the company's experimental drug for treatment ofDuchenne muscular dystrophy (DMD). Summit is an Oxford, United Kingdom, drug discovery company.

The award was made through MDA's Venture Philanthropy (MVP) arm, a part of MDA's translational research program.

Podcast Explores Blocking NF-kappa B in DMD

A December 2011 podcast from Nationwide Children's Hospital in Columbus, Ohio, explores how an inhibitor of a protein called NF-kappa B has been beneficial in a mouse model of Duchenne muscular dystrophy (DMD) and is now being developed as a potential DMD treatment.

The podcast is part of a Nationwide Children's series called "This Month in Muscular Dystrophy."

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.

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