Duchenne Muscular Dystrophy (DMD)

MD Briefs: Corrected Stem Cells, Membrane Sealants

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.

Heart Drug Being Tested in DMD

Eplerenone, a drug commonly used to prevent scarring after a heart attack, is being tested in a phase 2-3 clinical trial to determine whether it can stop or slow heart damage in people with Duchenne muscular dystrophy (DMD).  

Eplerenone blocks scarring that over time can turn heart muscle into nonfunctioning fatty tissue.

DMD/BMD — Zolt Arany

MDA awarded $352,188 to Zolt Arany, assistant professor in medicine at Beth Israel Deaconess Medical Center, part of Harvard Medical School in Boston, for research into the role of skeletal muscle metabolism in Duchenne muscular dystrophy (DMD).

DMD/BMD — Vihang Narkar

Vihang Narkar, assistant professor at the University of Texas Health Science Center at Houston, was awarded $302,326 to study the potential therapeutic value of increasing the overall amount of a specific type of muscle called "aerobic muscle" in Duchenne muscular dystrophy (DMD).

Loss of the structural protein dystrophin, the underlying cause of DMD, causes muscle instability, damage and progressive weakness, accompanied by a decrease in the ability to produce energy, along with severe fatigue.

DMD/BMD — Steve Wilton

MDA awarded a research grant totaling $368,100 to professor Steve Wilton at the University of Western Australia in Perth, for continued research into a strategy called "exon skipping," which bypasses mutations in the dystrophin gene responsible for Duchenne muscular dystrophy (DMD).

DMD/BMD — Sean Forbes

MDA awarded $179,327 to scientific researcher Sean Forbes at the University of Florida in Gainesville for research into impaired blood flow to muscles lacking the dystrophin protein in Duchenne muscular dystrophy (DMD).

DMD/BMD — Michele Calos

Michele Calos, professor in the department of genetics at Stanford University School of Medicine in Stanford, Calif., received a $200,000 MDA research grant to develop a new stem cell-based therapy for Duchenne muscular dystrophy (DMD).

"Funding from MDA is critical for us to develop these new ideas that could lead to novel strategies to combat DMD," Calos said. "A prior MDA grant has allowed us to make progress in the studies, and we are now poised to perform the critical experiments."

DMD/BMD — Louis Kunkel

MDA awarded a grant totaling $375,000 to Louis Kunkel, director of the program in genomics at Children's Hospital in Boston, for research into compounds already approved for human use that may alter disease progression in Duchenne muscular dystrophy (DMD).

DMD/BMD — Josephine Nalbantoglu

Josephine Nalbantoglu, associate professor in the department of neurology and neurosurgery at McGill University in Montreal, Canada, received an MDA grant totaling $313,170 for research into increasing levels of the muscle protein utrophin as a therapeutic strategy in Duchenne muscular dystrophy (DMD).

DMD/BMD — Gordon Lynch

MDA awarded $375,000 to Gordon Lynch, professor of physiology at the University of Melbourne, Australia, for research into strategies aimed at improving muscle function in Duchenne muscular dystrophy (DMD).

Lynch's research builds on previous studies conducted by his group and others that have demonstrated the potential of proteins called "growth factors" to preserve muscle fibers. These growth factors are regulated by a family of proteins called insulin-like growth factor binding proteins (IGFBPs).

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