Lysosomal Storage Diseases
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Sign up freeTop drugs studied for this disease
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CLINICAL_TRIALS trial_result · 2026-08-06Clinical Trial Results: A Fabry Disease Gene Therapy Study [Phase 1, Phase 2]
Phase 1/2 trial results for a Fabry disease gene therapy were posted after termination of the study.
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CLINICAL_TRIALS trial_result · 2026-04-07Clinical Trial Update: Study of Inborn Errors of Cholesterol Synthesis and Related Disorders
This clinical trial update notes the completed status of a study on inborn errors of cholesterol synthesis and related disorders, including …
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CLINICAL_TRIALS trial_result · 2023-11-14Clinical Trial Update: Nutritional Therapy in Late-onset Pompe Disease [Phase 2]
Phase 2 trial evaluating nutritional therapy for late-onset Pompe disease with muscle loss; current status is unknown according to ClinicalT…