Lysosomal Storage Diseases
📧 Sign up to get email alerts when something new happens for Lysosomal Storage Diseases.
Sign up freeTop drugs studied for this disease
-
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.
-
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 …
-
CLINICAL_TRIALS research · 2026-01-15Clinical Trial Update: Identification and Characterization of Novel Non-Coding Variants That Contribute to Genetic Disorders
This completed clinical trial update from Duke University describes identifying and characterizing novel non-coding genetic variants that co…
-
CLINICAL_TRIALS research · 2025-09-11Clinical Trial Update: A Pilot Study for Systematic Neonatal Screening for Lysosomal Storage Diseases Using Tandem Mass Spectrometry [NA]
A pilot study evaluating systematic neonatal screening for lysosomal storage diseases using tandem mass spectrometry is currently recruiting…
-
CLINICAL_TRIALS research · 2024-11-18Clinical Trial Update: Overview: Use of Respiratory Support At Home in Children
Overview of a planned study on home respiratory support for children with various conditions; the trial is not yet recruiting.
- Want full summaries, importance scores, and drug timelines? Upgrade to Plus or Pro.
-
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…
-
CLINICAL_TRIALS research · 2019-01-22Clinical Trial Update: Cellular Pharmacodynamics of Small Molecules in Lysosomal Storage Disorders
Clinical trial update on cellular pharmacodynamics of small molecules in lysosomal storage diseases; sponsor is Lysosomal and Rare Disorders…
-
CLINICAL_TRIALS withdrawal · 2017-06-29Clinical Trial Update: Fetal Umbilical Cord Blood (UCB) Transplant for Lysosomal Storage Diseases [Phase 1]
Phase 1 fetal umbilical cord blood transplant trial for lysosomal storage diseases has been withdrawn.