FDA Approves New Therapies for MPS IIIA and SMA | The CheckRare Brief Ep 8 by CheckRare Staff| Published on: Sep 23, 2026

This week we discuss the FDA’s approval of Isembyld for SMA, the approval of a gene therapy for MPS IIIA, and the FDA’s Center for Devices and Radiological Health new Rare Disease Impact Initiative. https://checkrare.com/fda-approves-new-therapies-for-mps-iiia-and-sma-the-checkrare-brief-ep-8/