Clinical Trials & Studies of Neuromuscular Disease - Selective Listing

Active Trials | Completed Trials | Postponed Trials | Discontinued Trials

Clinical trials are experiments conducted using human participants that seek to determine the value of a potential treatment, such as a drug, dietary supplement or exercise program. Most trials compare a treated group of participants with a "control," or comparison, group, which receives an inert substance ("placebo") or a sham treatment.

Many trials are divided into phases, the first of which is usually quite small and almost always designed only to assess the safety of the new treatment and how well it's tolerated. Phases 2 and 3 of a trial are larger and address questions of effectiveness and dose.

The term "study" implies either a trial or a more general research question under investigation, such as how many people with a certain set of symptoms have a particular genetic change or how parents cope with raising a child with a disability.

To learn more about the purposes and risks of clinical trials, please go to www.clinicaltrials.gov. This site, sponsored by the National Institutes of Health, contains general information and listings of specific trials.

For specific information about children and clinical trials, see www.ChildrenAndClinicalStudies.nhlbi.nih.gov.

See also "Being a Co-Adventurer: Clinical trials Involve Risks, Rights, Responsibilities" in the May-June 2008 issue of Quest, MDA's bimonthly magazine.

The full names of the diseases in MDA's program and more
information about each disease can be found at http://www.mda.org/disease/.