MDA Applauds Expanded US FDA Approval of ELEVIDYS Gene Therapy for DMD Patients Ages 4 and Above

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Congenital Myasthenic Syndromes (CMS)


In the past, people with congenital myasthenic syndromes (CMS) were often told they had myasthenia gravis (MG) and were subjected to years of pointless immunosuppressive therapy.

By identifying the genetic defects that cause CMS, MDA-funded scientists have improved the diagnosis of CMS and discovered drugs that are effective against it.

MDA-supported scientists are continuing to probe the genetic and molecular underpinnings of the various congenital myasthenic syndromes so that specific treatments for these can be developed or refined.

Current projects include the development of a new mouse model of a congenital myasthenic syndrome; genetic analysis of a worm with a slow-channel myasthenic syndrome; studies of how the nerve-muscle junction forms; and overall methods to improve the diagnosis, treatment and prevention of congenital myasthenic syndromes.

For more, read the Quest article Managing Myasthenia.

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