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Latest Duchenne muscular dystrophy Stories

2014-03-26 08:29:20

Urges President to Support Safe and Effective Treatment for Duchenne Muscular Dystrophy WASHINGTON, March 26, 2014 /PRNewswire/ -- The Race to Yes, a national campaign by families with children facing death from Duchenne Muscular Dystrophy achieved an important milestone just after 8pm EDT last night when the 100,000th person signed their White House petition urging the U.S. Food and Drug Administration to approve a breakthrough drug that has been shown to halt the progress of the...

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...

2014-03-20 16:26:23

Leading Duchenne Organization Launches Certified Duchenne Care Center Program HACKENSACK, N.J., March 20, 2014 /PRNewswire-USNewswire/ -- Nationwide Children's Hospital was named the first Certified Duchenne Care Center by Parent Project Muscular Dystrophy (PPMD), the leading advocacy organization working to end Duchenne muscular dystrophy (Duchenne). PPMD is launching the Certified Duchenne Care Center Program as part of its robust Transforming Duchenne Care Initiative. Over the...

2014-03-14 08:27:16

CAMBRIDGE, Mass., March 14, 2014 /PRNewswire/ -- Catabasis Pharmaceuticals Inc. today announced that data from its Phase 1 clinical trial of SMART Linker conjugate CAT-1004 will be presented at the Muscular Dystrophy Association's 2014 Clinical Conference in Chicago, Ill. Catabasis' chief medical officer, Joanne Donovan, M.D., Ph.D., will discuss Duchenne muscular dystrophy (DMD) animal model data and data from a Phase 1 clinical trial during the poster session from 6:00 to 8:00 p.m. CDT on...

2014-03-04 12:27:01

Parents Pushing FDA for Accelerated Approval of Safe and Effective Drug WASHINGTON, March 4, 2014 /PRNewswire-iReach/ -- After calling on the FDA directly to approve a breakthrough therapy for Duchenne Muscular Dystrophy, and appealing to Congress on behalf of the thousands of children facing this fatal genetic disease, concerned families have launched a national campaign and petition drive called "The Race to Yes" at TheRacetoYes.org to underscore the urgent need for accelerated...

2014-02-24 08:28:11

Annual Advocacy Conference Focuses on Accelerating FDA Review And Enacting MD-CARE Act Update into Law WASHINGTON, Feb. 24, 2014 /PRNewswire-USNewswire/ -- More than 100 advocates seeking to end Duchenne muscular dystrophy (Duchenne) are converging on Washington for the annual Parent Project Muscular Dystrophy (PPMD) Advocacy Conference to urge Congressional action on key legislation and to help accelerate the Food and Drug Administration's (FDA) review of candidate Duchenne...

2014-02-19 12:29:18

HACKENSACK, N.J., Feb. 19, 2014 /PRNewswire-USNewswire/ -- DuchenneConnect, a program of Parent Project Muscular Dystrophy (PPMD), is thrilled to announce receiving a funding award of $970,000 by the Patient-Centered Outcomes Research Institute (PCORI) to take part in developing a health data network. DuchenneConnect will be part of PCORnet: the National Patient-Centered Clinical Research Network. The DuchenneConnect network is one of 29 networks that were approved for PCORnet. The...

2014-02-10 20:21:08

Call on FDA for Accelerated Approval of Promising Duchenne Muscular Dystrophy Treatment WASHINGTON, Feb. 10, 2014 /PRNewswire-iReach/ -- Health staffers from over 80 Congressional offices together with agency and industry officials jammed a briefing room last Friday to hear mothers, clinicians and leading researchers call on the FDA to accept and promptly review a new drug application for eteplirsen, a promising new treatment for Duchenne Muscular Dystrophy that could save thousands...

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...


Word of the Day
omphalos
  • The navel or umbilicus.
  • In Greek archaeology: A central boss, as on a shield, a bowl, etc.
  • A sacred stone in the temple of Apollo at Delphi, believed by the Greeks to mark the 'navel' or exact center-point of the earth.
'Omphalos' comes from the ancient Greek.
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