Becker Muscular Dystrophy - BMD

In this second of two fall reports on treatment development for Duchenne muscular dystrophy, news about ARM210, CAP-1002, PDE5 inhibitors, drisapersen and CAT-1004 is presented

posted on December 4, 2014 - 3:13pm
Several experimental drugs are in development to treat Duchenne muscular dystrophy (DMD), a genetic disorder that results in a lack of the dystrophin protein in cardiac and skeletal muscle cells.

Genetically corrected human stem cells help mice with a disease resembling limb-girdle MD; cell membrane sealants are being studied in Duchenne and other forms of muscular dystrophy

posted on July 3, 2012 - 11:49am
LGMD2D mice benefit from corrected human stem cells A multinational team of scientists successfully transplanted genetically corrected muscle stem cells derived from people with type 2D limb-girdle muscular dystrophy (LGMD2D) into LGMD2D research mice and saw better muscle function in these mice than in similar mice that didn't receive the cells.

New drug trials in Becker and Duchenne muscular dystrophies are open, and a new strategy to improve Pompe disease treatment is proposed

posted on June 28, 2012 - 2:11pm
PTC begins non-US study of ataluren in DMD/BMD

The fourth annual BMD conference will be held in Chicago and live-streamed online; registration deadline is Aug. 1

posted on June 25, 2012 - 6:00am
Update (Sept. 7, 2012): Videos of all presentations from the BMD Conference have been archived on MDA's BMD Conference video page. For a brief synopsis of each video, see BMD Conference Videos Cover Health Care, Research and Daily Living.

A study of 22 couples considering preimplantation genetic diagnosis (PGD) has revealed a four-phase decision-making process

posted on May 11, 2012 - 3:59pm
Decision making about preimplantation genetic diagnosis (PGD) is a complex, multiphase process for couples, a new study has found. Understanding it, the investigators say, may be helpful to prospective parents who know they're at risk for transmitting a genetic disorder, and to the professionals who advise them.

Findings on the immune response to dystrophin in Duchenne and Becker MD, treatment of cardiomyopathy in DMD, and eteplirsen in DMD were presented

posted on April 26, 2012 - 4:41pm
Update (June 1, 2012) ­—  The brief Drugs help DMD-related cardiomyopathy has been updated to include a May 2012 podcast provided by Nationwide Children's Hospital's "This Month in Muscular Dystrophy."

'Best practices' for treating various neuromuscular diseases are described in this third and final report on MDA's 2012 Clinical Conference

posted on April 19, 2012 - 6:00am
Several experts presented their views of "best practices" for care of people with neuromuscular disorders at MDA's 2012 Clinical Conference, held in Las Vegas March 4-7. Many questions remain about optimal care in these disorders, but it's clear that attention to heart and respiratory function are of paramount importance. This article looks at:

In this second report on MDA's Clinical Conference, updates on experimental therapies in development for six neuromuscular diseases are described

posted on April 6, 2012 - 6:00am
The progress of several experimental therapies currently in development for neuromuscular diseases was discussed at MDA's 2012 Clinical Conference, held in Las Vegas March 4-7.