Clinical Trial Update: Efficacy and Safety of Human Lipoprotein Lipase (LPL)[S447X] Expressed by an Adeno-Associated Viral Vector in LPL-deficient Subjects [Phase 2, Phase 3]

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Summary

Completed Phase 2/3 trial evaluating the efficacy and safety of LPL S447X gene therapy expressed by an adeno-associated viral vector in individuals with lipopro…

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Diseases / indications

• Familial Chylomicronemia Syndrome
• Lipoprotein Lipase Deficiency

Drugs mentioned

• Glybera (alipogene tiparvovec)

Snippet

Sponsor: Amsterdam Molecular Therapeutics | Conditions: Familial Lipoprotein Lipase Deficiency | Phase 2, Phase 3 | Status: COMPLETED