Lysosomal Storage Disease
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CLINICAL_TRIALS trial_result · 2025-10-01Clinical Trial Update: High Dose Inspiratory Muscle Training in LOPD [NA]
Clinical trial update on high-dose inspiratory muscle training for late-onset Pompe disease, a lysosomal storage disorder, with the study st…
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CLINICAL_TRIALS research · 2017-07-02Clinical Trial Update: The Classification and Cause of Leukodystrophies of Unknown Cause
This clinical trial update discusses how leukodystrophies of unknown cause are classified and what is known about their underlying causes. T…
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CLINICAL_TRIALS trial_result · 2016-10-11Clinical Trial Update: Reduced-Intensity Hematopoietic Stem Cell Transplant for High Risk Lysosomal and Peroxisomal Disorders [NA]
Completed trial evaluating reduced-intensity hematopoietic stem cell transplantation for high-risk lysosomal storage diseases and peroxisoma…
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CLINICAL_TRIALS trial_result · 2014-08-13Clinical Trial Update: Reduced Intensity Conditioning for Umbilical Cord Blood Transplant in Pediatric Patients With Non-Malignant Disorders [Phase 1]
Clinical trial update on reduced intensity conditioning for umbilical cord blood transplantation in pediatric patients with non-malignant di…