Five Questions with SMA Researcher Chris Lorson

Chris Lorson, professor of veterinary pathology, and molecular biology and immunology at the University of Missouri in Columbia, was awarded an MDA research grant totaling $300,000 over three years to optimize an approach currently in clinical trials for spinal muscular atrophy (SMA) by delivering an enhanced form of drug combined with other potential synergistic therapies. . . .

Read More

SMA – Kevin Foust, Ph.D.

Kevin Foust, assistant professor in the department of neuroscience at Ohio State University in Columbus, was awarded an MDA research grant totaling $293,378 over three years to investigate disruption of gut bacteria in spinal muscular atrophy (SMA). The gut microbiome is the collection of organisms that inhabit a healthy gastrointestinal (GI) tract. Dysbiosis, or changes . . .

Read More

SBMA – Diane Merry, Ph.D.

Diane Merry, associate professor at Thomas Jefferson University in Philadelphia, was awarded an MDA research grant totaling $300,000 over three years to identify therapeutic opportunities to promote normal androgen receptor function while preventing the toxic effects of polyglutamine expansion in spinal-bulbar muscular atrophy (SBMA). Working with GlaxoSmithKline, Merry will test small molecule compounds that activate . . .

Read More

Muscle disease – Colin Crist, Ph.D.

Colin Crist, an assistant professor in the department of human genetics at McGill University in Québec, Canada, was awarded an MDA research grant totaling $300,000 over three years to investigate a biological mechanism that determines whether skeletal muscle stem cells, which play an important role in muscle regeneration, self-renew or differentiate. Crist has identified inhibitors . . .

Read More

McArdle disease – Ronald Haller, M.D.

Ronald Haller, professor of neurology and neurotherapeutics at University of Texas Southwestern Medical Center in Dallas, was awarded an MDA research grant totaling $300,000 over three years to identify the cause and possible effective treatment for the oxidative defect that accompanies blocked glycogen breakdown in McArdle disease (phosphorylase deficiency). Haller plans to assess levels of . . .

Read More

LGMD – Criss Hartzell, Ph.D.

Criss Hartzell, professor of cell biology at Emory University School of Medicine in Atlanta, Ga., was awarded an MDA research grant to elucidate the mechanisms underlying type 2L limb-girdle muscular dystrophy (LGMD), caused by mutations in the ANO5 gene. Hartzell will examine the role the ANO5 protein plays in trafficking and fusion of cell membranes . . .

Read More

FSHD – Antoine de Morrée, Ph.D.

Antoine de Morrée, a postdoctoral scholar at Palo Alto Veterans Institute for Research and Stanford University in Palo Alto, Calif., was awarded an MDA development grant totaling $180,000 over three years to test a way to stop muscles from making toxic DUX4 protein as a possible treatment for facioscapulohumeral muscular dystrophy (FSHD). The goals of de . . .

Read More

FA – Sanjay Bidichandani, M.B.B.S., Ph.D.

CMRI Claire Gordon Duncan Chair in Genetics and Professor of Pediatrics Sanjay Bidichandani, at University of Oklahoma Health Sciences Center in Oklahoma City, was awarded an MDA research grant totaling $300,000 over three years to address clinically and scientifically important questions regarding the use of existing and novel HDAC inhibitors to increase levels of the . . .

Read More

DMD – Thomas Rando, M.D., Ph.D.

Thomas Rando, at Palo Alto Veterans Institute for Research and Stanford University in California, was awarded an MDA research grant totaling $300,000 over three years to develop a mouse model — a so-called “reporter mouse” — that will reflect and quantify degeneration of skeletal muscles. Importantly, scientists will be able to use the mouse to . . .

Read More

DMD – Donghoon Lee, Ph.D.

Donghoon Lee, a research associate professor in the department of radiology at the University of Washington in Seattle, was awarded an MDA research grant totaling $300,000 over three years to develop imaging biomarkers for Duchenne muscular dystrophy (DMD). Lee will develop noninvasive magnetic resonance imaging (MRI) and magnetic resonance spectroscopy (MRS) methodologies that reflect specific . . .

Read More