NIH

A drug that has FDA approval for other indications is being tested in CCD patients who are at least 7 years old

posted on February 13, 2015 - 11:50am
The National Institute of Nursing Research (NINR) at the National Institutes of Health in Bethesda, Md., is conducting a study to determine if an antioxidant drug that has been approved by the U.S. Food and Drug Administration for other indications can reduce the severity of some symptoms in people with central core disease (CCD), a genetic disease caused by defects in a structure known as...

A drug that has FDA approval for other indications is being tested in CCD patients who are at least 7 years old

posted on February 13, 2015 - 11:49am
The National Institute of Nursing Research (NINR) at the National Institutes of Health in Bethesda, Md., is conducting a study to determine if an antioxidant drug that has been approved by the U.S. Food and Drug Administration for other indications can reduce the severity of some symptoms in people with central core disease (CCD), a genetic disease caused by defects in a structure known as...

A drug that has FDA approval for other indications is being tested in CCD patients who are at least 7 years old

posted on February 13, 2015 - 11:48am
The National Institute of Nursing Research (NINR) at the National Institutes of Health in Bethesda, Md., is conducting a study to determine if an antioxidant drug that has been approved by the U.S. Food and Drug Administration for other indications can reduce the severity of some symptoms in people with central core disease (CCD), a genetic disease caused by defects in a structure known as...

A panel of experts explored issues related to newborn screening for Duchenne muscular dystrophy

posted on September 19, 2012 - 12:49pm
Update (Feb. 8, 2013): Longtime MDA grantee Jerry Mendell, who directs the Center for Gene therapy at Nationwide Children's Hospital in Columbus, Ohio, published Report of MDA Muscle Disease Symposium on Newborn Screening for Duchenne Muscular Dystrophy in Muscle & Nerve, Feb. 8, 2013.

Participants will be randomly assigned to a strength or stretching exercise program, and undergo multiple evaluations

posted on August 9, 2011 - 12:00am
Update June 19, 2013: This study is nearing completion. However, enrollment is still open and additional participants are needed.

International Rare Disease Day and a few proposed laws, policies and government activities of interest to the MDA community

posted on February 28, 2011 - 8:58am
International Rare Disease Day 2011 Monday, February 28, 2011, marks the fourth annual International Rare Disease Day, in which hundreds of patient organizations from more than 40 countries conduct awareness-raising activities around the slogan “Rare but Equal.” The worldwide event is an effort to draw attention to rare diseases and the millions of people who are affected by them — including all...

The National Institutes of Health has awarded more than $4.5 million to specialized muscular dystrophy research centers at three U.S. institutions

posted on September 30, 2010 - 3:47pm
The U.S. National Institutes of Health (NIH) announced Sept. 29, 2010, that it will allocate more than $4.5 million for the first year of a five-year commitment to explore new treatment strategies for various forms of muscular dystrophy. Support will go to three U.S. institutions: Nationwide Children's Hospital in Columbus, Ohio; the University of Pennsylvania in Philadelphia; and the University...

An update on stem cell research related to neuromuscular disease as of July 2009

posted on July 1, 2009 - 5:17pm
QUEST Vol. 16, No. 3
Stem cells — immature cells with the potential to develop into different tissue types — have been heralded as a major advance for developing treatments for a variety of diseases. That’s true for diseases of the nerves and muscles, where such cells could potentially be transplanted into the body and either support or replace a patient’s ailing cells.