HIGHLIGHTS
- who: John W. Day from the (UNIVERSITY) have published the paper: Advances and limitations for the treatment of spinal muscular atrophy, in the Journal: (JOURNAL)
- what: The aim of this manuscript is to orient physicians to the evolving landscape of SMA treatment.
SUMMARY
Approved therapies for treating SMA Nusinersen was approved in the U.S. in 2016 and the E.U. in 2017 for treating patients with SMA of all ages with 5q SMA based on the results of two phase 3 clinical trials. TOPAZ demonstrated the potential clinical benefit of . . .
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