Latest muscular dystrophy Stories

PTC Therapeutics Closes $60 Million Private Financing

2013-03-07 12:27:23

-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...

Parent Project Muscular Dystrophy Funds UCLA Project to Identify Genetic Changes that Affect the Severity of Duchenne Muscular Dystrophy

2013-03-01 12:25:07

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:...

Parent Project Muscular Dystrophy Marks Rare Disease Day with Multiple Awareness Events

2013-02-28 08:29:39

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...

Advocates in Muscular Dystrophy Community Press Congress for Fast Action on Muscle Disease Research and Treatment

2013-02-14 20:24:37

TUCSON, Ariz., Feb. 14, 2013 /PRNewswire-USNewswire/ -- The Muscular Dystrophy Association, Parent Project Muscular Dystrophy and the Foundation to Eradicate Duchenne today called on the U.S. Senate and the U.S. House of Representatives to reauthorize the MD CARE Act of 2001, and to continue federal support for the accelerated pace of research and treatment development for muscular dystrophy. The nonprofits also are urging the public to contact their U.S. Senators and ask them to vote...

Catabasis Announces Positive Phase 1 Data for CAT-1004 Validating the SMART Linker Technology Platform

2013-02-11 08:25:55

CAMBRIDGE, Mass., Feb. 11, 2013 /PRNewswire/ -- Catabasis Pharmaceuticals, Inc., today announced that a three-part series of Phase 1 studies showed that CAT-1004, a SMART Linker conjugate of docosahexaenoic acid (DHA) and salicylate created using the company's proprietary technology, was safe and well tolerated. Data also show that the conjugated compound was absorbed following oral administration and metabolized to its active components intracellularly, providing proof of concept...

Experimental Gene Therapy Treatment Offers Hope For Youngster With Duchenne Muscular Dystrophy

2013-02-09 09:05:38

UC Davis Health System [ Watch The Video ] Jacob Rutt is a bright 11-year-old who likes to draw detailed maps in his spare time. But the budding geographer has a hard time with physical skills most children take for granted ― running and climbing trees are beyond him, and even walking can be difficult. He was diagnosed with a form of muscular dystrophy known as Duchenne when he was two years old. The disease affects about 1 in 3,500 newborns ― mostly boys ― worldwide. It...

MDF Awards $200,000 for Research into Treatments for Myotonic Dystrophy

2013-02-07 16:26:03

MENLO PARK, Calif., Feb. 7, 2013 /PRNewswire-USNewswire/ -- The Myotonic Dystrophy Foundation (MDF) has awarded two $100,000 grants to postdoctoral Fellows working in universities to encourage basic research in the management, treatment and cure of myotonic dystrophy (DM). Each of the 2013-2014 recipients will receive $50,000 a year for two years. (Logo: http://photos.prnewswire.com/prnh/20130207/DC56674LOGO) This award cycle brings MDF's total research funding to over $1.5M and...

11-Time Olympic Medalist Ryan Lochte Plunges Into Advocacy

2013-02-07 12:26:14

Swimmer Travels to Capitol Hill to Urge Congress to Continue Support of People with Duchenne Muscular Dystrophy WASHINGTON, Feb. 7, 2013 /PRNewswire-USNewswire/ -- Parent Project Muscular Dystrophy (PPMD), the leading non-profit in the United States focused entirely on Duchenne muscular dystrophy (Duchenne), welcomes 11-time Olympic medalist Ryan Lochte to their Annual Advocacy Conference in Washington, D.C., February 10-12, 2013. Lochte, one of the top American performers at the...

Parent Project Muscular Dystrophy Funds Collection and Analysis of Imaging Data from Eteplirsen Study

2013-02-01 16:26:11

University of Florida to Receive $59,000 Grant from Duchenne-specific Organization HACKENSACK, N.J., Feb. 1, 2013 /PRNewswire-USNewswire/ -- Parent Project Muscular Dystrophy (PPMD) announced that it has funded $59,000 to Krista Vandenborne, PhD of the University of Florida in Gainesville to test the ability of magnetic resonance imaging (MRI) to detect changes in the muscles of those living with Duchenne muscular dystrophy (Duchenne) who participated in the Sarepta phase II...

Parent Project Muscular Dystrophy Grants $175,000 for Biomarkers, Non-Ambulatory Endpoints in Duchenne Muscular Dystrophy

2013-01-18 08:23:43

Dr. Craig McDonald of UC Davis to Receive Supplemental Funds HACKENSACK, N.J., Jan. 18, 2013 /PRNewswire-USNewswire/ -- Parent Project Muscular Dystrophy (PPMD) has awarded Dr. Craig McDonald of the University of California, Davis (UC Davis) $175,000 in supplemental funds to expand his ongoing study through the 20 CINRG (Cooperative International Neuromuscular Research Group) centers to better understand the progression of Duchenne muscular dystrophy (Duchenne) and determine the...