Becker Muscular Dystrophy - BMD

In a May 22, 2014, letter, Prosensa provides updates on drisapersen and other Duchenne MD compounds in its pipeline, and on its natural history study

posted on May 28, 2014 - 8:12am
Dutch biopharmaceutical company Prosensa, developer of the experimental Duchenne muscular dystrophy (DMD) drug drisapersen, says it is moving the compound forward through regulatory agencies in the U.S. and Europe and will resume administering it to a first group of trial participants in the third quarter of 2014.

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.

In this first of several reports on MDA's Clinical Conference, the role of genetics and immunology in different neuromuscular diseases is described

posted on March 19, 2012 - 3:08pm
More than 500 physicians, allied health care professionals and MDA staff attended the MDA's 2012 Clinical Conference in Las Vegas, March 4-7. The program emphasized:

The shorter-than-normal dystrophin protein produced via an experimental exon-skipping drug allows proper localization of proteins at the muscle-fiber membrane

posted on February 8, 2012 - 2:45pm
Shortened versions of the muscle protein dystrophin — produced by skipping a section of genetic instructions called exon 51 — appear to be functional, says a new report from the United Kingdom.

MDA has awarded 38 grants totaling more than $12 million to support research into general muscle health and more than 15 neuromuscular diseases in its program

posted on February 1, 2012 - 10:00am
The Muscular Dystrophy Association has awarded 38 new grants totaling more than $12 million to fund research projects focused on its continuing mission to uncover the causes of, and develop therapies for, the more than 40 neuromuscular diseases in its program. MDA's Board of Directors reviewed and approved the new grants based on recommendations from the Association's Scientific and Medical...