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

Latest News in Cell and Gene Therapy for Rare Disease Day 2024
Latest News in Cell and Gene Therapy for Rare Disease Day 2024

February 29th 2024

In observance of Rare Disease Day, held this year on February 29, catch up on some of the latest data updates from clinical trials for rare diseases.

Around the Helix: Cell and Gene Therapy Company Updates – February 28, 2024
Around the Helix: Cell and Gene Therapy Company Updates – February 28, 2024

February 28th 2024

Heather Lau, MD, MS
UX111 Reduces Heparin Sulfate, Correlating With Benefit in Cognitive Function in Pediatric MPSIIIA

February 28th 2024

CGTLive’s Weekly Rewind
CGTLive’s Weekly Rewind – February 23, 2024

February 23rd 2024

Around the Helix: Cell and Gene Therapy Company Updates – February 21, 2024
Around the Helix: Cell and Gene Therapy Company Updates – February 21, 2024

February 21st 2024

Video Interviews
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