Lysosomal Disorders
The CGTLive™ Lysosomal Disorders specialty topic page contains video interviews with key opinion leaders discussing the most relevant FDA actions, clinical guideline updates, and clinical trial findings related to cell therapies, gene therapies, and engineered and regenerative medicines developed for Lysosomal storage diseases. It also houses the most up-to-date clinical news coverage in the field of gene and cell therapy for diseases related to lysosomal storage and dysfunction.
Latest News
February 19th 2025
The PDUFA action date for the BLA has been set at August 18, 2025, and no advisory committee meeting is currently planned.
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