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 a trial, SMA gene therapy EXG001-307 helped a small group of young children with type 1 better control their heads and sit ...

In the 1987 science fiction spoof movie “Spaceballs,” Princess Vespa (Daphne Zuniga) is betrothed to Prince Valium (Jim J.