Hope Through Research

At MDA, we take a big picture perspective across the full spectrum of neuromuscular diseases to uncover scientific and medical breakthroughs that accelerate treatments and cures. The power in our research approach is that we can often apply learnings from one disease to progress in others to bring urgently-needed answers to our families.

Emery-Dreifuss Muscular Dystrophy (EDMD)

It is very difficult to develop specific therapies for very rare forms of muscular dystrophy such as Emery-Dreifuss Muscular Dystrophy (EDMD). Howard Worman, professor of medicine and pathology and cell biology at Columbia University in New York, has been developing compounds to treat the disease, specifically the problems in the heart. These are in preclinical development.

In addition, potential therapeutics in development for other muscular dystrophies (if successful) also may be effective for these diseases. Strategies that are based on the type of mutation that cause the disease may help some patients. For example, ataluren, a nonsense mutation suppressing drug that MDA has supported may help patients with this type of mutation.

Other strategies, such as muscle strengthening drugs, also may be effective in many muscular dystrophies. MDA also has supported researchers who have worked out the genetic causes of EDMD, and this is a starting point for more specific therapies.

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