Muscular Dystrophies

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.

Man with CMD Died Doing What He Loved

Michael Wogan had been looking forward to the trip to the National Championship Air Races in Reno, Nev.

According to his younger brother James, “Michael liked to get out and travel, and he was so excited about getting on a plane.”

Michael’s older brother Billy, 26 — who, like Michael, 22, and James, 19, has congenital muscular dystrophy (CMD)— was supposed to attend the show with their dad. When he couldn’t, Michael went instead.

He promised to tell his brothers all about it.

Induced Stem Cells Require Cautious Approach

Stem cells have been much in the news lately, including for neuromuscular diseases.

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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