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Last updated on April 16, 2014 at 1:21 EDT

Latest Dystrophin Stories

2014-03-21 23:01:12

The John Owen Adventure Dodgeball event is for children and adults alike to raise money and awareness for the fatal disease, Duchenne Muscular Dystrophy. North Royalton, OH (PRWEB) March 21, 2014 John Owen's Adventure Inc. is thrilled to launch the second annual Play for JOA-Dodgeball event bringing together children and adults alike to enjoy this classic sport while fundraising to cure the fatal disease Duchenne Muscular Dystrophy (DMD). "Our 2013 Tourney was a huge...

2014-01-24 08:23:47

SOUTH PLAINFIELD, N.J., Jan. 24, 2014 /PRNewswire/ -- PTC Therapeutics, Inc. (NASDAQ: PTCT) (PTC) today announced that the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) has adopted a negative opinion on the Company's marketing authorization application (MAA) for conditional approval of ataluren for the treatment of nonsense mutation Duchenne muscular dystrophy (nmDMD). The response from the CHMP is consistent with the Company's previous...

2013-12-12 16:25:26

Program Trains Researchers in Drug Development TUCSON, Ariz., Dec. 12, 2013 /PRNewswire-USNewswire/ -- The Muscular Dystrophy Association today announced it has awarded a second grant under the auspices of its Bridge-to-Industry (B2I) program. B2I is designed to train promising researchers in translational research by providing experience in both academia and the biopharmaceutical industry. Translational research refers to the process of "translating" laboratory findings into...

2013-11-14 08:34:37

NEWTON, Mass. and TUCSON, Ariz., Nov. 14, 2013 /PRNewswire-USNewswire/ -- Halo Therapeutics LLC and the Muscular Dystrophy Association (MDA) today announced that $500,000 has been awarded for development of the experimental drug HT-100 as a treatment for Duchenne muscular dystrophy (DMD). The award to Halo, a subsidiary of DART Therapeutics Inc., comes from MDA Venture Philanthropy (MVP), part of MDA's translational research program. (Logo:...

2013-09-18 12:55:59

A preclinical study led by researchers at Children’s National Medical Center has found that a new oral drug shows early promise for the treatment of Duchenne muscular dystrophy (DMD). The results, published in EMBO Molecular Medicine, show that the drug, VBP15, decreases inflammation and protects and strengthens muscle without the harsh side effects linked to current treatments with glucocorticoids such as prednisone. Duchenne muscular dystrophy results in severe muscle degeneration and...

2013-08-26 08:25:20

-Two publications further validate the use of 6MWT as a primary endpoint in Duchenne Muscular Dystrophy (DMD) clinical trials- SOUTH PLAINFIELD, N.J., Aug. 26, 2013 /PRNewswire/ -- Data published in the medical journal Muscle & Nerve demonstrate the clinical meaningfulness of the 6-minute walk test (6MWT) as a primary endpoint to measure disease progression and walking ability in ambulatory Duchenne muscular dystrophy (DMD) trials. The research also showed that a range of 20 to...

2013-08-14 12:26:57

Leading Duchenne Organization Announces Request for Applications Focused on the Development of Oligos HACKENSACK, N.J., Aug. 14, 2013 /PRNewswire-USNewswire/ -- Parent Project Muscular Dystrophy (PPMD) - the nation's leading voice for patients and families impacted by Duchenne muscular dystrophy (Duchenne) - announced that it will fund up to $1 million in projects by mid-2014 focused on the development of antisense oligonucleotides or other techniques to skip exons that are not...

2013-07-15 12:25:15

DART's Phase 1b/2a Study to Determine Safety and Tolerability of Lead Drug Candidate, Help Validate New Endpoint for Future DMD Studies CAMBRIDGE, Mass., July 15, 2013 /PRNewswire/ --DART Therapeutics Inc., an innovative, new-model biotechnology firm focused on developing therapies for Duchenne muscular dystrophy (DMD), has initiated a phase 1b/2a study of its lead drug candidate, HT-100 (delayed-release halofuginone). The phase 1b study (with a six-month 2a extension) in...

2013-07-08 12:13:59

Children with Duchenne muscular dystrophy often die as young adults from heart and breathing complications. However, scientists have been puzzled for decades by the fact that laboratory mice bearing the same genetic mutation responsible for the disease in humans display only mild symptoms and no cardiac involvement. Now, researchers at the Stanford University School of Medicine have developed a mouse model that accurately mimics the course of the disease in humans. The study is the first...

Promise In Duchenne Muscular Dystrophy Shown With Genetic Editing
2013-06-05 08:48:09

Duke University Using a novel genetic 'editing' technique, Duke University biomedical engineers have been able to repair a defect responsible for one of the most common inherited disorders, Duchenne muscular dystrophy, in cell samples from Duchenne patients. Instead of the common gene therapy approach of adding new genetic material to "override" the faulty gene, the Duke scientists have developed a way to change the existing mutated gene responsible for the disorder into a normally...