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Lysosomal Storage Disease
Prenatal Enzyme Therapy Could Help Treat Rare Genetic Disease
2022-11-21
Takeda Embarks On a Billion-Dollar Gene Therapy Deal with Selecta Biosciences
2021-10-08
Venglustat’s Phase 2/3 Trial Failure Pushes Sanofi to Drop Kidney Disease Program
2021-06-02
Investigational Gene Therapy for Cystinosis Obtains FDA’s Orphan Drug Designation
2020-03-10
Rare Disease Biotech, Azafaros Finds Big Money in Small Molecules, Raises US$ 28M in Series A
2020-02-12
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