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Expert talks about Spinal Muscular Atrophy

With the approval of the first drug for Spinal Muscular Atrophy in December 2016, called Spinraza, the neuropediatrician Alexandra Prufer, a specialist in this degenerative disorder, spoke with Portal Health Connections about the history of the disease and the evolution of the studies for the development of effective treatments. Prufer explained how the new drug works in the patient's system, detailed the research that resulted in the drug's approval and talked about the different approaches to curing the disease currently under investigation. Check out the video (in Portuguese).


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