Gene Therapy Well-Tolerated in Late-Onset Pompe DiseaseByAbby ReinhardFebruary 13th 2022Interim safety data from the FORTIS study were presented at WORLDSympsoium.
Patient Dies in Phase 1b Mini-Dystrophin Gene Therapy Trial in DMD ByAbby ReinhardDecember 21st 2021The FDA has placed the investigational new drug application for the treatment on clinical hold.