Duchenne Muscular Dystrophy (DMD)

Sarepta Reaffirms Commitment to Eteplirsen, Other DMD Drugs

BMD: Follistatin Gene Transfer Results Encouraging

Results from a trial involving injection of genes for the follistatin protein into the thigh muscles of both legs in six men with Becker muscular dystrophy (BMD) show the experimental treatment appears safe and and was associated with improvement in walking ability (distance walked in six minutes) in four of the six trial participants.

DMD - Darek Gorecki, Ph.D.

Darek Gorecki, a professor of molecular medicine at the University of Portsmouth in the United Kingdom, has been awarded an MDA research grant totaling $253,800 over three years to study whether blocking a specific receptor (molecular docking site) that enhances inflammation can be beneficial to muscle fibers affected by Duchenne muscular dystrophy (DMD). He will conduct experiments in mice.

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

DMD - Nicoletta Filigheddu, Ph.D.

Nicoletta Filigheddu, a molecular biologist at the University of Piemonte Orientale Amedeo Avogadro in Italy, has been awarded an MDA research grant totaling $253,800 over three years to investigate whether a hormone called ghrelin can improve muscle regeneration or the success of muscle stem cell transplantation in mice with a disorder mimicking Duchenne muscular dystrophy (DMD).

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

DMD - Federica Montanaro, Ph.D.

Federica Montanaro, Ph.D., a principal investigator at the Center for Gene Therapy at Nationwide Children's Hospital in Columbus, Ohio, and an assistant professor of pediatrics at Ohio State University, was awarded an MDA research grant totaling $253,800 over three years to study how decreased activity in a biochemical signaling pathway called "hedgehog" affects muscle stem cells during muscle repair. She will find out whether increasing hedgehog signaling in mice with a DMD-like disorder preserves muscle function.

DMD - Dongsheng Duan, Ph.D.

Dongsheng Duan, a professor in the Department of Molecular Microbiology & Immunology at the University of Missouri in Columbia, has received an MDA research infrastructure grant totaling $169,200 over two years to develop and maintain a national canine tissue bank so that investigators studying Duchenne muscular dystrophy (DMD) will have access to normal and dystrophin-deficient  tissue samples taken from dogs. Dystrophin is the muscle protein that is missing in DMD patients.

DMD - Da-Zhi Wang, Ph.D.

Da-Zhi Wang, an associate professor of pediatrics at Boston Children's Hospital and Harvard Medical School, was awarded an MDA research grant totaling $253,800 over three years to study the role of a compound called miR-155, a "microRNA," in muscle function and regeneration and to see whether reducing it could help treat Duchenne muscular dystrophy (DMD). Wang has found that mice with lacking miR-155 have better muscle function and regeneration than mice that have this substance.

DMD - Terence Partridge, Ph.D.

Terence Partridge, a professor at Children’s Research Institute at Children’s National Medical Center in Washington, D.C., was awarded an MDA research grant totaling $253,800 over three years to develop and test a new type of compound that causes exon skipping – a way for cells to make functional proteins from flawed genes -- as a potential treatment for Duchenne muscular dystrophy (DMD).  Current exon-skipping drugs for DMD have limited, if any, ability to enter heart muscle cells.

DMD - Erica Reeves, Ph.D.

Erica Reeves, a biologist and vice president of research and operations at ReveraGen BioPharma, was awarded an MDA translational research grant totaling of $1,015,200 over one year to conduct the first human trial of VBP15, an experimental drug in development  to treat Duchenne muscular dystrophy (DMD).  This first trial will be in healthy volunteers.

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

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