Not a disease but a genetic disorder that might occur even as a fresh mutation.


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  • Clinical Trials

    Facilitating access to clinical trials and research updates


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Clinical Trials

A clinical trial is a test in humans to evaluate biomedical or health-related outcomes, including experimental medications or therapies. Clinical trials are experiments, not treatments, and participation requires careful consideration.

Although it's possible to benefit from participating in a clinical trial, it's also possible that no benefit — or even harm — may occur. Keep Muscular Dystrophy Foundation India informed about any clinical trial participation. (Note that MDFINDIA has no ability to influence who is chosen to participate in a clinical trial.)

Clinical trials are conducted in a series of successive steps, called phases. Each phase is designed to answer a separate research question.

  • Observational: Tests health or biomedical outcomes to understand the disease course. These studies are conducted without an intervention (e.g., an investigational new drug).
  • Phase 0: Tests how a new drug distributes in the body at a very low dose (below what is expected to have a therapeutic benefit).
  • Phase 1: Tests whether a new drug is safe in a small group of people, and determines the best way to dose it.
  • Phase 2: Tests whether a new drug is effective in a medium-sized group of people, and confirms its safety.
  • Phase 3: Tests whether a new drug is effective in a large group of people, and monitors its side effects and safety.
  • Phase 4: Studies the drug after it has been marketed to understand the benefits and side effects associated with long-term use.

*Note: Some clinical trials are not assigned a phase by the U.S. Food and Drug Administration (FDA). These include trials that test devices or behavioral interventions.

Current trials in DMD: Please visit the link for a complete list of clinical trials in DMD worldwide http://www.treat-nmd.eu/research/clinical-research/overview-current-trials-dmd/current-trials/

Current trials in LGMD: The following link lists all LGMD trials registered or updated on clinicaltrials.gov in the last 1000 days. http://www.treat-nmd.eu/research/clinical-research/lgmd-current-trials/

Current trials in Spinal Muscular Atrophy: The following link lists all LGMD trials registered or updated on clinicaltrials.gov in the last 1000 days. http://www.treat-nmd.eu/research/clinical-research/sma-current-trials/

Trials for all other Muscular Dystrophies: Please type the name of the muscular dystrophy in the search panel of the link to find out ongoing trials for each type of muscular dystrophy. https://clinicaltrials.gov/ct2/search/index