United by Myotonic Dystrophy, a Daughter Honors her Mom on Mother’s Day — and the 25th Anniversary of Her Death

In the decades since Leslie Krongold’s mother died at the age of 56, she has learned that they likely shared a neuromuscular disease — myotonic dystrophy — and, as Leslie approaches her own 56th birthday next year,  she feels more committed than ever to defying the odds her my mother couldn’t.

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Spinraza a Breakthrough for SMA

Emflaza

On Dec. 23, 2016 the FDA approved Spinraza for the treatment of SMA. In clinical trials to test Spinraza, participants who received treatment with the drug experienced life-changing outcomes they wouldn’t have been expected to achieve.

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