Mucopolysaccharidosis Type
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CLINICAL_TRIALS trial_result · 2024-10-22Clinical Trial Update: Longitudinal Study of Bone Disease in Children with Mucopolysaccharidoses (MPS) I, II, and VI
This clinical trial update reports on a longitudinal study of bone disease in children with mucopolysaccharidoses types I, II, and VI. The s…
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CLINICAL_TRIALS trial_result · 2024-08-06Clinical Trial Update: Efficacy and Safety of YW17 (Laronidase-CinnaGen) Compared to Aldurazyme® in MPS I Patients [Phase 3]
A Phase 3 clinical trial comparing YW17 (Laronidase-CinnaGen) with Aldurazyme in patients with mucopolysaccharidosis type I has completed, e…
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CLINICAL_TRIALS trial_result · 2024-07-25Clinical Trial Update: A Clinical Study Evaluating the Safety, Tolerability, and Initial Efficacy of JWK008 in Patients With Mucopolysaccharidosis Type I [Phase 1]
A Phase 1 clinical trial evaluating the safety, tolerability, and initial efficacy of JWK008 in patients with Mucopolysaccharidosis Type I i…
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CLINICAL_TRIALS trial_result · 2024-05-03Clinical Trial Update: Long Term Follow-up (LTFU) of Subjects Who Received SB-318, SB-913, or SB-FIX
Long-term follow-up study update for subjects treated with Sangamo's SB-318, SB-913, or SB-FIX, evaluating ongoing outcomes in hemophilia B …
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CLINICAL_TRIALS trial_result · 2023-01-26Clinical Trial Update: Ascending Dose Study of Genome Editing by the Zinc Finger Nuclease (ZFN) Therapeutic SB-318 in Subjects With MPS I [Phase 1, Phase 2]
Phase 1/2 dose-escalation study of ZFN genome editing (SB-318) in MPS I was terminated; results posted in 2023.
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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 …
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CLINICAL_TRIALS trial_result · 2017-12-13Clinical Trial Update: Parental Coping With Challenging Behavior in Mucopolysaccharidosis Type I-III
A clinical trial examining parental coping with challenging behavior in children with mucopolysaccharidosis types I–III has completed.
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CLINICAL_TRIALS trial_result · 2017-09-15Clinical Trial Update: Effects of Adalimumab in Mucopolysaccharidosis Types I, II and VI [Phase 1, Phase 2]
Clinical trial evaluating adalimumab in mucopolysaccharidosis types I, II, and VI has completed phases 1 and 2 with results posted in 2017.
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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…
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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…
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CLINICAL_TRIALS trial_result · 2013-02-21Clinical Trial Update: Intrathecal Enzyme Replacement Therapy for Spinal Cord Compression in Mucopolysaccharidosis (MPS) I [Phase 1]
A Phase 1 clinical trial evaluating intrathecal enzyme replacement therapy for spinal cord compression in Mucopolysaccharidosis type I was t…
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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…
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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…
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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…
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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.