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
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
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
Update (Sept. 7, 2012): Videos of all presentations from the BMD Conference have been archived on MDA's BMD Conference video page.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
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
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
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
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.