Latest Duchenne muscular dystrophy Stories
HACKENSACK, N.J., April 29, 2013 /PRNewswire-USNewswire/ -- Parent Project Muscular Dystrophy (PPMD), the leading nonprofit in the United States focused entirely on Duchenne muscular dystrophy (Duchenne), launched the Duchenne Central mobile app, in collaboration with Siren Interactive, a digital relationship marketing agency focused on rare disorder therapies. The app, which is available as a free download for Apple and Android devices, is an easy-to-use and on-the-go tool to locate...
Makes Recommendations to Speed Responsible Access to New Therapies for Duchenne Muscular Dystrophy, Other Rare Neurological Disorders HACKENSACK, N.J., April 23, 2013 /PRNewswire-USNewswire/ -- Parent Project Muscular Dystrophy (PPMD) has released Putting Patients First, a white paper outlining recommendations to speed responsible access to new therapies for Duchenne muscular dystrophy (Duchenne) and other, rare, serious, and life-threatening neurological disorders. PPMD developed...
Conference to include data from Sarepta, GSK/Prosensa on exon-skipping trials for Duchenne dystrophy, plus updates on many other potential therapies TUCSON, Ariz., April 22, 2013 /PRNewswire-USNewswire/ -- Some 500 members of the scientific and medical research community are scheduled to attend the 2013 MDA Scientific Conference, "Therapy Development for Neuromuscular Diseases: Translating Hope into Promise," April 21-24 in Washington, D.C., according to representatives of the...
TARRYTOWN, N.Y. and TUCSON, Ariz., April 17, 2013 /PRNewswire/ -- ARMGO Pharma and the Muscular Dystrophy Association (MDA) today announced that $1 Million has been awarded for preclinical work in support of an Investigational New Drug (IND) application with the U.S. Food and Drug Administration (FDA) for ARM210, a novel, orally available, small-molecule Rycal drug that has potential as a treatment for Duchenne muscular dystrophy (DMD). The funding award to ARMGO Pharma comes from...
CHARLOTTESVILLE, Virginia and CAMBRIDGE, Massachusetts, March 26, 2013 /PRNewswire/ -- DART Therapeutics Inc., an innovative, new-model biotechnology firm focused on developing therapies for Duchenne muscular dystrophy (DMD), and Biovista, a leader in systematic drug repositioning, announced today that they have entered into a research collaboration to identify and develop novel drug repositioning candidates for DMD using Biovista's Clinical Outcome Search Space (COSS)(TM)...
HACKENSACK, N.J., March 21, 2013 /PRNewswire-USNewswire/ -- Parent Project Muscular Dystrophy (PPMD) has awarded University of Minnesota researcher Dr. Rita Perlingeiro a grant for $220,000 to develop a therapeutic approach for Duchenne muscular dystrophy (Duchenne) using a new type of "adult-derived" stem cell called "induced pluripotent stem cells" or "iPSCs." iPSCs can be created from non-controversial sources like skin, have the ability to multiply indefinitely, and can be...
SARM Could Represent a New Category of Therapy for Duchenne Muscular Dystrophy, Potential for Other Neuromuscular Diseases CAMBRIDGE, Mass., March 20, 2013 /PRNewswire/ -- DART Therapeutics Inc., an innovative, new-model biotechnology firm focused on developing therapies for Duchenne muscular dystrophy (DMD), announced today that it is developing a SARM drug candidate obtained from Belgium-based Galapagos NV. In early studies, the drug candidate, renamed DT-200, demonstrated...
-Funds to support Phase 3 confirmatory trials of ataluren- SOUTH PLAINFIELD, N.J., March 7, 2013 /PRNewswire/ -- PTC Therapeutics, Inc. (PTC) today announced the successful completion of a $60 million financing led by Brookside Capital Partners Fund, L.P. Joining Brookside as new investors in this financing are Adage Capital Management, Jennison Associates L.L.C. (on behalf of fund clients), Longwood Fund and additional top-tier institutional investors. In conjunction with the...
HACKENSACK, N.J., March 1, 2013 /PRNewswire-USNewswire/ -- Parent Project Muscular Dystrophy (PPMD) will provide $100,000 to Dr. Stanley Nelson of the University of California, Los Angeles, to examine the genomes of 40 boys with Duchenne muscular dystrophy (Duchenne) who are either very mildly affected or very severely affected. The goal is to identify changes in genes other than dystrophin that may affect the course of the disease. (Logo:...
HACKENSACK, N.J., Feb. 28, 2013 /PRNewswire-USNewswire/ -- Parent Project Muscular Dystrophy (PPMD), the leading non-profit in the United States focused entirely on Duchenne muscular dystrophy (Duchenne), will mark the six year anniversary of Rare Disease Day today by participating in two important events that will raise awareness about Duchenne and share with audiences compelling stories from our community. (Logo: http://photos.prnewswire.com/prnh/20100119/DC39975LOGO) PPMD...
