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2014-09-09 08:31:34

-Top-line data expected H2 2015- SOUTH PLAINFIELD, N.J., Sept. 9, 2014 /PRNewswire/ -- PTC Therapeutics, Inc. (NASDAQ: PTCT) today announced that it has completed enrollment of ACT DMD, the Phase 3 confirmatory trial of Translarna(TM) (ataluren) for patients with nonsense mutation Duchenne muscular dystrophy (nmDMD). Top-line data from the trial is expected in the second half of 2015 and will support further approvals globally, following European approval received earlier this...

2014-07-29 12:34:51

Bill will help ensure law focuses adequately on needs of adults with Duchenne and other forms of muscular dystrophy WASHINGTON, July 29, 2014 /PRNewswire-USNewswire/ -- The House of Representatives today approved legislation to update the landmark Muscular Dystrophy Community Assistance, Research, and Education (MD-CARE) Act so the law continues getting results for all patients impacted by Duchenne and other forms of muscular dystrophy....

2014-07-09 16:28:13

-French ATU Cohort and Other Named Patient Programs Authorized - SOUTH PLAINFIELD, N.J., July 9, 2014 /PRNewswire/ -- PTC Therapeutics, Inc. (NASDAQ: PTCT) today announced the initiation of a reimbursed expanded access program (EAP). PTC's EAP program is intended to make Translarna(TM) (ataluren) available to patients before commercial availability in certain countries. Where mechanisms exist and in accordance with local regulations, PTC will make Translarna available to nonsense...

2014-06-09 12:30:50

HACKENSACK, N.J., June 9, 2014 /PRNewswire-USNewswire/ -- Parent Project Muscular Dystrophy (PPMD) - the leading patient advocacy organization fighting to end Duchenne muscular dystrophy (Duchenne) - announced that a recent treatment preferences and benefit/risk study led by the organization has been published in Clinical Therapeutics. The article entitled, "A Community-Engaged Approach to Quantifying Caregiver Preferences for the Benefits and Risks of Emerging Therapies for Duchenne...

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...


Word of the Day
grass-comber
  • A landsman who is making his first voyage at sea; a novice who enters naval service from rural life.
According to the OED, a grass-comber is also 'a sailor's term for one who has been a farm-labourer.'