Clinical Trial Update: Gene Therapy for IGHMBP2-Related Diseases [Phase 1, Phase 2]

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Summary

Ongoing Phase 1/2 gene therapy trial for IGHMBP2-related diseases (SMARD1 and Charcot-Marie-Tooth disease type 2S) is currently enrolling by invitation.

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Diseases / indications

• Charcot-marie-tooth Disease Type 2s
• Spinal Muscular Atrophy with Respiratory Distress Type

Snippet

Sponsor: Megan Waldrop | Conditions: SMARD1, CMT2S | Phase 1, Phase 2 | Status: ENROLLING_BY_INVITATION