Rare Diseases Report 2022

Spinal muscular atrophy: Patient care in the age of genetically targeted therapy


 

References

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12. Pascual SI et al. Ongoing phase 2/3 DEVOTE (232SM203) randomized, controlled study to explore high-dose nusinersen in SMA: Part A interim results and Part B enrollment update. Presented at MDA Clinical and Scientific Conference 2021, Mar 15-18.

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