Jerry R. Mendell, MD, has been named to the TIME100 Health list for his contributions to the treatment of neuromuscular ...
A range of genetic mutations associated with spinal muscular atrophy (SMA) were linked to abnormal activation of the immune ...
In the 1987 science fiction spoof movie “Spaceballs,” Princess Vespa (Daphne Zuniga) is betrothed to Prince Valium (Jim J. Bullock), who dozes off constantly. He has chronic drowsiness, so much so ...
In a trial, SMA gene therapy EXG001-307 helped a small group of young children with type 1 better control their heads and sit ...
SMN-K186R, a modified version of the SMN protein that is deficient in people with spinal muscular atrophy (SMA), was more effective than normal SMN when delivered by gene therapy, a mouse study ...
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