Duchenne Muscular Dystrophy (DMD)

DMD: Eteplirsen Data Still Encouraging After Three Years

Boys with Duchenne muscular dystrophy (DMD) caused by specific genetic mutations who were treated with the experimental drug eteplirsen for 168 weeks (3.2 years) continued to show an advantage in the distance they were able to walk compared to those treated with the drug for only 144 weeks (2.8 years), although all boys had declined in walking distance since the start of this phase 2b trial of eteplirsen.

PTC Begins Submission of New Drug Application for DMD Stop Codon Read-Through Drug

New Jersey-based PTC Therapeutics has begun submitting a rolling new drug application (NDA) to the U.S. Food and Drug Administration (FDA) for the approval of its experimental drug for the treatment of Duchenne muscular dystrophy (DMD) caused by specific flaws (mutations) in the dystrophin gene.

Myostatin Blocker To Be Tested in DMD

Multinational pharmaceutical comany Pfizer recently opened a phase 2 study of an experimental compound that may be beneficial in Duchenne muscular dystrophy (DMD) through its ability to block myostatin, a naturally occurring protein that is known to limit muscle growth.

DMD: Utrophin Modulator SMT C1100 To Undergo Further Testing

SMT C1100, an experimental compound designed to boost levels of the potentially therapeutic protein utrophin in patients with Duchenne muscular dystrophy (DMD), will undergo further testing in boys with this disorder along with dietary specifications that may improve its absorption by the body, its developer has announced.

Sarepta Reaffirms Commitment to Eteplirsen, Other DMD Drugs

Cambridge, Mass.-based Sarepta Therapeutics, developer of eteplirsen and other "exon-skipping" drugs designed  to treat Duchenne muscular dystrophy (DMD), provided overall reassurance about eteplirsen's development and some details of future plans to the DMD community in a Dec.

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.