Rare Pediatric Disease and Fast Track Designations Granted to Gene Therapy for Myotubular Myopathy – Rare Disease Report
Rare Disease Report |
Rare Pediatric Disease and Fast Track Designations Granted to Gene Therapy for Myotubular Myopathy
Rare Disease Report XLMTM is the result of mutations in the MTM1 gene, which encodes the myotubularin protein. The myotubularin protein plays a significant part in the development, maintenance and function of cells in the skeletal muscles. AT132 contains an AAV8 vector … Audentes Therapeutics Announces Rare Pediatric Disease and Fast Track Designations for AT132 for the Treatment of … Audentes Therapeutics (BOLD) Receives Rare Pediatric Disease and Fast Track Designations for AT132 for Treatment … |
