Muscular Dystrophies

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).

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."

International MMD Consortium Includes Professionals, Families

The 8th International Myotonic Dystrophy Consortium Meeting (IDMC-8), was an exciting mix of the latest scientific developments and clinical research in types 1 and 2 myotonic dystrophy (MMD1 and MMD1, also known as DM1 and DM2).

MMD Research Update Streamed for Families Dec. 3

As part of the upcoming International Myotonic Dystrophy Consortium (IDMC-8) in Clearwater, Fla., a session will be held Saturday, Dec. 3, 2011, to provide a research update for individuals and families affected by myotonic muscular dystrophy (MMD).

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.

Jerry Mendell's Research Team Receives Award

A team headed by neurologist Jerry Mendell, a longtime MDA research grantee and director of the MDA Clinic at Nationwide Children's Hospital in Columbus, Ohio, has received the prestigious Annals of Neurology prize for an outstanding contribution to clinical neuroscience.

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