Sarepta Therapeutics and Clinigen Launch Managed Access Program for Duchenne Muscular Dystrophy Patients

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Sarepta Therapeutics (Cambridge, MA), biopharmaceutical company focused on the discovery and development of unique RNA-targeted therapeutics for the treatment of rare neuromuscular diseases, and Clinigen Group's Idis Managed Access division, have initiated a Managed Access Program (MAP) for eteplirsen in certain geographies to treat eligible Duchenne muscular dystrophy (DMD) patients amenable to exon 51 skipping.

The MAP (also known as an early/expanded access, or named patient program) provides a mechanism through which physicians can legally and ethically prescribe eteplirsen to patients who meet pre-specified medical criteria and where funding can be secured, it is reported.

Initially, the program is being launched in select countries within Europe, North America and South America for certain patients where eteplirsen is not currently approved. Sarepta plans to expand the program to include more countries over time.

The program will be administered by Clinigen Group's Idis Managed Access division.


For more information on the MAP, visit