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Latest Duchenne muscular dystrophy Stories

2014-10-20 12:30:49

CLEVELAND and COLUMBUS, Ohio, Oct. 20, 2014 /PRNewswire-USNewswire/ -- Data published today in the journal Molecular Therapy demonstrates initial clinical proof of principle of a gene therapy to increase muscle strength and function in Becker muscular dystrophy patients. The paper, titled "A Phase I/IIa Follistatin Gene Therapy Trial for Becker Muscular Dystrophy" describes results of the dose ascending trial to assess safety and efficacy in six patients with Becker muscular...

2014-09-26 16:24:06

Bipartisan Legislation Enacted Unanimously by Congress will Strengthen and Update Landmark MD-CARE Act WASHINGTON, Sept. 26, 2014 /PRNewswire-USNewswire/ -- Capping a two-year advocacy campaign led by Parent Project Muscular Dystrophy (PPMD), today President Barack Obama signed the MD-CARE Act Amendments of 2014 into law, providing a significant victory to the Duchenne and broader muscular dystrophy communities. http://photos.prnewswire.com/prnvar/20100119/DC39975LOGO The law makes...

2014-09-17 16:26:57

Fourth Clinic to Receive Certification from Leading Duchenne Organization HACKENSACK, N.J., Sept. 17, 2014 /PRNewswire-USNewswire/ -- Seattle Children's Hospital was named a Certified Duchenne Care Center by Parent Project Muscular Dystrophy (PPMD), the leading advocacy organization working to end Duchenne muscular dystrophy (Duchenne). Seattle Children's is the fourth center to be certified by PPMD, recognizing the hospital's dedication to improving care for people living with...

2014-09-17 12:24:23

WACO, Texas, Sept. 17, 2014 /PRNewswire-USNewswire/ -- For the sixth year in a row, college football coaches nationwide will join together in support of the Coach To Cure MD program, which will be held during games of Saturday, September 27, 2014. The rapidly growing annual effort has raised more than a million dollars to battle Duchenne muscular dystrophy. On September 27th, American Football Coaches Association (AFCA) members will wear a Coach To Cure MD logo patch on the sidelines...

2014-09-17 08:29:28

SOUTH PLAINFIELD, N.J., Sept. 17, 2014 /PRNewswire/ -- PTC Therapeutics, Inc. (NASDAQ: PTCT) today announced the expansion of its global presence with the addition of key executives to leadership positions in PTC's global commercial team and the establishment of its international headquarters in Dublin, Ireland. Significant pre-launch activities are well underway as PTC prepares for the commercial launch of Translarna(TM) (ataluren) in the European Union. In August of this year,...

2014-09-10 12:28:50

However, Less Than One-Third of Duchenne Patients Are in Subpopulations That Will Benefit From New Disease-Modifying Therapies, According to Findings from Decision Resources Group BURLINGTON, Mass., Sept. 10, 2014 /PRNewswire/ -- Decision Resources Group finds that the first three emerging disease-modifying therapies (DMTs)--PTC Therapeutics' Translarna (ataluren), Prosensa's drisapersen and Sarepta's eteplirsen--will likely receive regulatory approval for Duchenne muscular dystrophy (DMD) in...

2014-09-09 12:28:23

MDA Veteran Brings 25 Years of Experience Working with Muscular Dystrophy HACKENSACK, N.J., Sept. 9, 2014 /PRNewswire-USNewswire/ -- Pat Furlong, Founding President and CEO of Parent Project Muscular Dystrophy (PPMD), the leading advocacy organization working to end Duchenne muscular dystrophy (Duchenne), announced today that Annie Kennedy has joined the PPMD team as Senior Vice President of Legislation and Public Policy. Ms. Kennedy joins PPMD after being with the Muscular Dystrophy...

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...

2014-09-07 23:02:18

Coalition Duchenne supports World Duchenne Awareness Day Newport Beach, California (PRWEB) September 07, 2014 California based charity Coalition Duchenne supports the First World Duchenne Awareness Day on the 7th of September 2014. The 7th of the 9th has been chosen to represent the 79 exons in the Dystrophin gene. Duchenne Charities around the world will raise awareness for boys and young men who have Duchenne muscular dystrophy. Parents and supporters of Duchenne organizations will...

2014-08-31 20:20:38

Rocker donates $10,000 and promises 9-year-old ambassador a Harley ride CHICAGO, Aug. 31, 2014 /PRNewswire-USNewswire/ -- Viewers tuning into the 49(th) MDA Show of Strength Telethon are in for "Nothin' But a Good Time" when legendary rock star and reality TV star Bret Michaels covers American classic "Sweet Home Alabama" on the nationally televised show airing tonight on ABC television stations across the country. http://photos.prnewswire.com/prnvar/20140831/141734 In...


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The word 'bellycheer' may come from 'belle cheer', "good cheer".
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