Hurler Syndrome
📧 Sign up to get email alerts when something new happens for Hurler Syndrome.
Sign up freeTop drugs studied for this disease
-
CLINICAL_TRIALS trial_result · 2026-01-07Clinical Trial Update: MT2013-31: Allo HCT for Metabolic Disorders and Severe Osteopetrosis [Phase 2]
Phase 2 trial update describing allogeneic hematopoietic cell transplantation for metabolic disorders and severe osteopetrosis, with the stu…
-
CLINICAL_TRIALS trial_result · 2023-09-15Clinical Trial Update: Conditioning Regimen for Allogeneic Hematopoietic Stem-Cell Transplantation [Phase 1, Phase 2]
A Phase 1/2 clinical trial evaluating conditioning regimens for allogeneic hematopoietic stem-cell transplantation has completed recruitment…
-
CLINICAL_TRIALS trial_result · 2020-03-20Clinical Trial Update: Administration of IV Laronidase Post Bone Marrow Transplant in Hurler [Phase 1]
Phase 1 trial evaluating intravenous laronidase administration after bone marrow transplant in Hurler syndrome has completed with results po…
-
CLINICAL_TRIALS trial_result · 2019-07-11Clinical Trial Update: Stem Cell Transplant w/Laronidase for Hurler [Phase 2]
Phase 2 clinical trial updating stem cell transplant with Laronidase for Hurler syndrome (Mucopolysaccharidosis I) has completed enrollment …
-
CLINICAL_TRIALS trial_result · 2017-05-12Clinical Trial Update: Allogeneic Bone Marrow Transplant for Inherited Metabolic Disorders [Phase 2]
Phase 2 clinical trial update on allogeneic bone marrow transplant for inherited metabolic disorders; study completed with results posted in…
- Want full summaries, importance scores, and drug timelines? Upgrade to Plus or Pro.
-
CLINICAL_TRIALS trial_result · 2016-01-26Clinical Trial Update: A Study of Intrathecal Enzyme Therapy for Cognitive Decline in MPS I [NA]
Clinical trial assessing intrathecal enzyme therapy for cognitive decline in mucopolysaccharidosis type I has completed.
-
CLINICAL_TRIALS trial_result · 2014-05-05Clinical Trial Update: A Study Investigating the Relationship Between the Development of Laronidase Antibody and Urinary GAG (Glycosaminoglycan) Levels in Aldurazyme® Treated Patients [Phase 4]
Phase 4 study investigating the relationship between anti-laronidase antibodies and urinary glycosaminoglycans in Aldurazyme-treated patient…
-
CLINICAL_TRIALS trial_result · 2013-02-21Clinical Trial Update: Extension Study of Intrathecal Enzyme Replacement Therapy for MPS I [Phase 1]
Extension study of intrathecal enzyme replacement therapy for Mucopolysaccharidosis Type I (Phase 1) has been terminated. The trial involved…
-
CLINICAL_TRIALS trial_result · 2011-07-13Clinical Trial Update: Hematopoietic Stem Cell Transplantation (HCT) for Inborn Errors of Metabolism [Phase 2]
Phase 2 trial update on hematopoietic stem cell transplantation for inborn errors of metabolism; trial terminated with results posted in 201…
-
CLINICAL_TRIALS trial_result · 2009-08-19Clinical Trial Update: Clinical Study of Aldurazyme in Patients With Mucopolysaccharidosis (MPS) I [Phase 3]
Phase 3 study of Aldurazyme in patients with mucopolysaccharidosis type I (Hurler and Hurler-Scheie) has completed, with results posted in 2…
-
CLINICAL_TRIALS trial_result · 2009-06-16Clinical Trial Update: A Study Evaluating the Safety and Pharmacokinetics of Aldurazyme® (Laronidase) in MPS I Patients Less Than 5 Years Old [Phase 2]
Phase 2 study evaluating the safety and pharmacokinetics of Aldurazyme (laronidase) in patients under 5 years old with mucopolysaccharidosis…
-
CLINICAL_TRIALS trial_result · 2009-06-16Clinical Trial Update: Phase 3 Extension Study of the Safety and Efficacy of Aldurazyme® (Laronidase) in Mucopolysaccharidosis I (MPS I) Patients [Phase 3]
This Phase 3 extension study evaluated the safety and efficacy of Aldurazyme (laronidase) in patients with Mucopolysaccharidosis I and has c…
-
CLINICAL_TRIALS trial_result · 2009-06-16Clinical Trial Update: A Dose-optimization Study of Aldurazyme® (Laronidase) in Patients With Mucopolysaccharidosis I (MPS I) Disease [Phase 4]
Phase 4 dose-optimization study of Aldurazyme (Laronidase) in patients with Mucopolysaccharidosis I completed with results posted in 2009.
-
CLINICAL_TRIALS trial_result · 2009-02-12Clinical Trial Update: Study of Aldurazyme® Replacement Therapy in Patients With Mucopolysaccharidosis I (MPS I) Disease [Phase 3]
Phase 3 study of Aldurazyme (laronidase) for mucopolysaccharidosis I has completed, with results posted in 2009.