Spinal Muscular Atrophy with Respiratory Distress Type
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CLINICAL_TRIALS research · 2025-09-30Clinical Trial Update: Gene Therapy for IGHMBP2-Related Diseases [Phase 1, Phase 2]
Ongoing Phase 1/2 gene therapy trial for IGHMBP2-related diseases (SMARD1 and Charcot-Marie-Tooth disease type 2S) is currently enrolling by…