Muscular Dystrophies

Muscle disease - Shihuan Kuang, Ph.D.

Shihuan Kuang, an associate professor of animal sciences at Purdue University in West Lafayette, Ind., has been awarded an MDA research grant totaling $253,800 over three years to investigate the use of low-oxygen conditioning of cells to improve stem cell-based therapies to treat muscular dystrophies.

Funding for this MDA research grant began May 1, 2014.

Muscle disease - Stephen Hauschka, Ph.D.

Stephen Hauschka, a professor of biochemistry at the University of Washington-Seattle, has been awarded an MDA research grant totaling $253,800 over three years to develop and test new “on-off” gene “switches,” also known as “regulatory cassettes,” to be used in gene transfer therapies to treat muscle disorders.

Funding for this MDA research grant began May 1, 2014.

Muscle disease - Mariah Baker, Ph.D.

Mariah Baker, a research associate at the University of Texas Health Science Center at Houston, has been awarded an MDA development grant totaling $152,073 over three years to study how the release of calcium molecules is triggered in muscle cells. Improved understanding of this process could have implications for treating a variety of muscle disorders.

Funding for this MDA research grant began May 1, 2014.

Drug Development for DMD: Fall 2014 Update

Drug development for Duchenne muscular dystrophy (DMD) is progressing on several fronts. Here are some updates as of early November 2014.

PTC is moving forward with ataluren

MD-CARE Act Congressional Briefing Accelerates Reauthorization Momentum

"The energy in the room was high, and the mood was optimistic," said Annie Kennedy, MDA's senior vice president of advocacy, speaking about the Congressional briefing on reauthorization of the MD-CARE Act that she attended and helped to organize. The briefing was co-hosted by MDA and other muscular dystrophy organizations in Washington, D.C., on Feb.

Nitric Oxide Drug Trial in Becker MD Now Open

A new drug trial is now under way at Cedars-Sinai Medical Center in Los Angeles for men with Becker muscular dystrophy (BMD) who meet study criteria.

MDA Awards $8.5 Million to 31 Neuromuscular Disease Research Projects

In its summer 2013 round of research grant awards, the Muscular Dystrophy Association aims to catalyze research progress in a dozen neuromuscular diseases, with an eye toward applying that knowledge to related muscle diseases, as well.

“A large number of our grants are investigating new therapeutic technologies,” notes Jane Larkindale, MDA's vice president of research. “These are 'platform' technologies, where successes can be transferred well beyond the specific disease in which they are developed and tested.”

CMD: Aiming Simultaneously at Two Biological Targets

Researchers at Boston University, supported in part by MDA, say their experimental two-pronged strategy for merosin-deficient congenital muscular dystrophy (MDC1A) was highly successful in a mouse model of this disease and should be further investigated as a potential treatment approach for patients.

Turning Myotonic Dystrophy On and Off in Cells

MDA grantee Matthew Disney is a current and former MDA grantee at the Scripps Research Institute in Jupiter, Fla. Disney's current MDA grant is focused on targeting toxic RNA in type 2 myotonic dystrophy (MMD2, or DM2).

DMD/BMD: Questions About Ataluren’s Mechanism

It's been widely accepted that the mechanism by which the experimental drug ataluren appears to benefit walking ability in Duchenne muscular dystrophy (DMD) and Becker muscular dystrophy (BMD) is that it causes "read-through" of premature stop codons — genetic instructions that cause cells to stop making a protein before the process is complete.

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