News

Biopharmaceutical company Prosensa announced that they will present at the 6th annual conference on RNA Therapeutics, which will take place between February 16 and 17, 2015 in London, and will discuss the latest developments of its investigational drug for the treatment of Duchenne muscular dystrophy. Prosensa, which earlier…

A group of local CITGO marketers in Wisconsin are celebrating a year of successfully supporting the Muscular Dystrophy Association (MDA). The story is yet one more example of the pride and commitment among those who advocate and fundraise for treating and eventually curing the disease. In 2014, the eight marketers,…

A new study entitled “Precise Correction of the Dystrophin Gene in Duchenne Muscular Dystrophy Patient Induced Pluripotent Stem Cells by TALEN and CRISPR-Cas9” reports an iPSC-based gene therapy that can correct the genetic defect underlying Duchenne Muscular Dystrophy. The study was published in the journal…

Sarepta Therapeutics Inc. has announced that it will host a webcast and conference call dedicated specifically to the Duchenne muscular dystrophy (DMD) community, as the company is dedicated to the development of novel RNA-based therapies for the treatment of the genetic and disabling disease. The webcast and conference will take place on Tuesday, December…

Newport Beach-based Coalition Duchenne has granted its 2014 Lotus Award to professor Rachelle Crosbie-Watson from the University of California, Los Angeles (UCLA) in recognition of her outstanding work in education, research, and awareness for Duchenne muscular dystrophy. The award also includes a $10,000 grant, which will be given to the Center for Duchenne Muscular Dystrophy at…

The Kennedy Krieger Institute in Baltimore, Maryland has been granted Certified Duchenne Care Center status by the Parent Project Muscular Dystrophy (PPMD) advocacy group. The institute is the fifth institution to receive the designation, which recognizes its dedication to the improvement of healthcare services for patients suffering from Duchenne muscular dystrophy. Along with the…

Sarepta Therapeutics, Inc. recently announced a new study to confirm the dosages of their lead drug eteplirsen as a new therapeutic for treating Duchenne muscular dystrophy-affected patients with a baseline 6-minute walk test score. Duchenne muscular dystrophy (DMD) is a form of muscular dystrophy that is caused by a mutation of the Dystrophin gene,…

Biopharmaceutical company PTC Therapeutics was honored with the Outstanding Innovation Award at The 2014 Rare & Orphan Advocacy & Research Awards. The prize was granted to PTC for their lead therapy Translarna, which has recently become the first treatment for Duchenne muscular dystrophy to be approved in the European…

The International Duchenne Alliance has announced that it will provide a $500,000 grant to researchers Jerry Mendell, M.D. and Milo Biotechnology to support their work in a first ever follistatin gene therapy trial for Duchenne muscular dystrophy. The study will be held at Mendell at Nationwide Children’s Hospital in Columbus, Ohio, and the…

A team of researchers recently presented their latest results showing how injecting cardiac stem cells in mouse models of Duchenne muscular dystrophy can reverse damage to the heart. These findings carry a new therapeutic promise to patients with muscular waisting disorders. The study was presented in the American Heart…