PTC Therapeutics, Inc. has just announced its novel protein restoration therapy Translarna™ (ataluren) is now available to patients with Duchenne Muscular Dystrophy (DMD) in Germany, which is the first European Union country to offer the drug. The first batch of shipments are expected to roll out this week.
A new preclinical trial revealed the efficacy and IND/CTA enabling studies with ARM210/S48168. ARMGO Pharma and Servier are set to initiate clinical trials for the treatment of Duchenne Muscular Dystrophy (DMD). The companies have been working on a drug called Rycals®. Rycals targets the Ryanodine Receptor (RyR), an intracellular calcium…
Somerset, N.J. based Catalent Pharma Solutions, specialists in efficient delivery technology and development of medical and health care product solutions is collaborating with Valerion Therapeutics, LLC of Concord, Massachusetts – whose corporate focus is on bio-therapeutics for orphan genetic disease development. The alliance will join forces to develop Valerion’s…
Parent Project Muscular Dystrophy (PPMD) has partnered with Swiss pharmaceutical company Santhera Pharmaceuticals to conduct a collaborative study on the benefits and risks of Duchenne muscular dystrophy therapies. The researchers, which will use data from Santhera’s phase 3 clinical trial of idebenone, will be focused on determining…
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…
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