Mucopolysaccharidosis
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CLINICAL_TRIALS research · 2026-03-04Clinical Trial Update: Physical Activity Intervention for MPS
Brunel University is planning a clinical trial to evaluate a physical activity intervention for mucopolysaccharidosis, which is currently no…
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CLINICAL_TRIALS research · 2026-02-27Clinical Trial Update: Mucopolysaccharidosis I (MPS I) Registry
This entry reports the completion of the Mucopolysaccharidosis I registry clinical trial sponsored by Genzyme. No specific results are provi…
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CLINICAL_TRIALS withdrawal · 2025-12-09Clinical Trial Update: A Study of ELAPRASE in Treatment-naïve Participants With Hunter Syndrome (Mucopolysaccharidosis [MPS] II) [Phase 4]
A Phase 4 trial of Elaprase (idursulfase) in treatment-naïve patients with Hunter syndrome has been withdrawn.
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CLINICAL_TRIALS trial_result · 2025-09-17Clinical Trial Update: China Post-marketing Surveillance (PMS) Study of Aldurazyme® [Phase 4]
Phase 4 post-marketing surveillance study of Aldurazyme (laronidase) for Mucopolysaccharidosis I has been completed in China.
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CLINICAL_TRIALS trial_result · 2025-08-28Clinical Trial Update: An Extension Study of JR-171-101 Study in Patients With Mucopolysaccharidosis Type I (MPS I) [Phase 1, Phase 2]
An extension study (JR-171-101) in patients with Mucopolysaccharidosis Type I has completed.
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CLINICAL_TRIALS research · 2025-08-22Clinical Trial Update: Nutritional Assessment in Patient of Mucopolysaccharide " [NA]
A clinical trial sponsored by Assiut University is evaluating nutritional assessment in patients with Mucopolysaccharidosis, focusing on mal…
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CLINICAL_TRIALS trial_result · 2025-03-19Clinical Trial Update: A Study of JR-171 in Patients With Mucopolysaccharidosis I [Phase 1, Phase 2]
JR-171, investigated for Mucopolysaccharidosis I, completed a Phase 1/2 trial and posted results on 2025-03-19.
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CLINICAL_TRIALS trial_result · 2024-10-30Clinical Trial Update: Long Term Follow Up Of Patients Who Have Received Gene Therapy Or Gene Marked Products
Long-term follow-up results are reported for patients who received gene therapy or gene-marked products across several conditions, including…
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CLINICAL_TRIALS research · 2024-07-03Clinical Trial Update: Long Term Follow-Up for RGX-111
This entry reports a long-term follow-up study for RGX-111 in mucopolysaccharidosis type I and indicates ongoing enrollment by invitation.
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CLINICAL_TRIALS trial_result · 2024-02-20Clinical Trial Update: A Study of the Effect of Aldurazyme® (Laronidase) Treatment on Lactation in Female Patients With Mucopolysaccharidosis I (MPS I) and Their Breastfed Infants [Phase 4]
Phase 4 trial update evaluating the effect of Aldurazyme (Laronidase) on lactation in female patients with MPS I and their breastfed infants…
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CLINICAL_TRIALS trial_result · 2023-10-31Clinical Trial Update: Extension Study of Intrathecal Enzyme Replacement for Cognitive Decline in MPS I [NA]
An extension study of intrathecal enzyme replacement for cognitive decline in Mucopolysaccharidosis I has been terminated; results posted in…
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CLINICAL_TRIALS trial_result · 2023-03-15Clinical Trial Update: Safety and Dose Ranging Study of Human Insulin Receptor MAb-IDUA Fusion Protein in Adults and Children With MPS I [Phase 1, Phase 2]
Phase 1/2 trial assessing safety and dose ranging of a Human Insulin Receptor MAb-IDUA fusion protein in adults and children with Mucopolysa…
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CLINICAL_TRIALS trial_result · 2023-03-15Clinical Trial Update: Extension Study Evaluating Long Term Safety and Activity of AGT-181 in Children With MPS I [Phase 1, Phase 2]
Extension study evaluating long-term safety and activity of AGT-181 in children with MPS I completed.
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CLINICAL_TRIALS trial_result · 2018-12-12Clinical Trial Update: Safety, Tolerability, Ascending Dose and Dose Frequency Study of rhHNS Via an IDDD in MPS IIIA Patients [Phase 1, Phase 2]
Clinical trial update for rhHNS via an IDDD in MPS IIIA patients reports safety and tolerability data across ascending dose and dose frequen…
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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…
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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.
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CLINICAL_TRIALS trial_result · 2014-11-24Clinical Trial Update: Clinical Trial of Growth Hormone in MPS I, II, and VI [Phase 2, Phase 3]
A clinical trial evaluating growth hormone for Mucopolysaccharidosis I and II (Phase 2/Phase 3) was terminated; results were posted in 2014.
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CLINICAL_TRIALS trial_result · 2014-07-08Clinical Trial Update: ALD-101 Adjuvant Therapy of Unrelated Umbilical Cord Blood Transfusion (UCBT) in Patients With Inherited Metabolic Diseases [Phase 3]
Phase 3 trial of ALD-101 as adjuvant therapy with unrelated umbilical cord blood transfusion in inherited metabolic diseases was terminated.
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CLINICAL_TRIALS trial_result · 2014-07-02Clinical Trial Update: Immune Tolerance Study With Aldurazyme® (Laronidase) [Phase 1, Phase 2]
This clinical trial update covers a Phase 1/Phase 2 immune tolerance study of Aldurazyme (Laronidase) in patients with mucopolysaccharidosis…
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CLINICAL_TRIALS research · 2013-05-24Clinical Trial Update: Study to Detect Unrecognized Mucopolysaccharidosis in Children Visiting Rheumatology, Hand or Skeletal Dysplasia Clinics
Clinical trial update indicating termination of a study designed to detect unrecognized mucopolysaccharidoses in children at rheumatology, h…
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CLINICAL_TRIALS research · 2013-03-01Clinical Trial Update: Non-invasive Assessment of Intraocular Pressure in MPS by Use of the Ocular Response Analyzer.
This clinical trial update describes a non-invasive method to assess intraocular pressure in patients with Mucopolysaccharidosis using the O…
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CLINICAL_TRIALS trial_result · 2012-10-16Clinical Trial Update: Paraspinal Stimulation in Treating MPS [Phase 3]
This document provides an update on a Phase 3 clinical trial evaluating paraspinal stimulation for mucopolysaccharidosis. The trial status i…
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CLINICAL_TRIALS trial_result · 2012-06-04Clinical Trial Update: Hematopoietic Stem Cell Transplantation for Mucopolysaccharidosis [Phase 2, Phase 3]
Clinical trial update on hematopoietic stem cell transplantation for mucopolysaccharidosis across Phase 2 and Phase 3. The current status is…
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CLINICAL_TRIALS trial_result · 2009-09-04Clinical Trial Update: Stem Cell Transplantation for Hurler [Phase 2]
A Phase 2 clinical trial evaluating stem cell transplantation for Hurler syndrome has completed, with results posted in 2009.
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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.