Duchenne Muscular Dystrophy (DMD)

FDA Accepts Sarepta NDA for Eteplirsen to Treat DMD

Background: Sarepta announced today that the U.S. Food and Drug Administration (FDA) has accepted its New Drug Application (NDA) for eteplirsen to treat Duchenne muscular dystrophy (DMD).

Eteplirsen is an “exon-skipping” drug that targets a section of DNA called exon 51, and may help up to 13 percent of Duchenne muscular dystrophy (DMD) patients.

DMD - Addolorata Pisconti, Ph.D.

Addolorata Pisconti, at the University of Liverpool, United Kingdom, was awarded an MDA research grant totaling $300,000 over three years to study how the environment established by fibrosis and inflammation affects the maintenance and regenerative properties of muscle stem cells in Duchenne muscular dystrophy (DMD). Pisconti will work to determine whether serine protease inhibitor proteins are involved in the DMD disease process.

Marathon Pharmaceuticals Begins New Drug Application Process for Deflazacort to Treat Duchenne Muscular Dystrophy

Following a pre-NDA meeting with the U.S. Food and Drug Administration (FDA), Marathon Pharmaceuticals has announced that it will begin the New Drug Application (NDA) process for deflazacort as a treatment for Duchenne muscular dystrophy (DMD).

Marathon expects to submit the NDA in the first quarter of 2016. If the application is approved, the company could make the drug commercially available in the United States in the first quarter of 2017.

DMD - James Ervasti, Ph.D.

James Ervasti, professor in the department of biochemistry, molecular biology & biophysics at the University of Minnesota in Minneapolis, was awarded an MDA research grant totaling $300,000 over three years to develop methods of identifying non-invasive biomarkers that can be measured in all patients across the entire spectrum of Duchenne muscular dystrophy (DMD) disease severity.

DMD - Natassia Vieira, Ph.D.

Natassia Vieira, an investigator at Biosciences Institute, University of São Paulo - São Paulo, Brazil, was awarded an MDA development grant totaling $180,000 to study a genetic mechanism that appears to compensate for mutations in the gene that causes Duchenne muscular dystrophy (DMD). Vieira will study the mechanism by which overexpression of a protein called Jagged1 protects dogs and fish carrying a DMD-causing mutation from exhibiting the typical symptoms and progression of the disease.

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).

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 test the ability of cell and gene therapies to alter the progression of Duchenne muscular dystrophy (DMD) and other muscular dystrophies.

DMD - DeWayne Townsend, D.V.M., Ph.D.

DeWayne Townsend, assistant professor at the University of Minnesota in Minneapolis, was awarded an MDA research grant totaling $300,000 over three years to study the role of oxygen in the Duchenne muscular dystrophy (DMD)-affected heart.

DMD - Yetrib Hathout, Ph.D.

Yetrib Hathout, associate professor in the department of integrative systems biology at Children’s National Medical Center in Washington, D.C., was awarded an MDA research grant totaling $300,000 over three years to develop a panel of molecular biomarkers, detectable in blood, for Duchenne muscular dystrophy (DMD). If successful, these biomarkers could help clinicians monitor progression of the disease and determine whether a drug or treatment is working.

DMD - Rebecca Willcocks, Ph.D.

Rebecca Willcocks, an adjunct research assistant scientist in the department of physical therapy at the University of Florida in Gainesville, has been awarded an MDA development grant totaling $171,422 over three years to study magnetic resonance biomarkers in Duchenne muscular dystrophy (DMD). Willcocks will use magnetic resonance imaging (MRI) and magnetic resonance spectroscopy (MRS) to describe how DMD affects the shoulder, upper arm and forearm muscles of boys, ages 9 to 18 years, with DMD.

Pages