Mucopolysaccharidosis Type
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Sign up freeTop drugs studied for this disease
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CLINICAL_TRIALS other · 2026-04-08Clinical Trial Update: Registry of Patients Diagnosed With Lysosomal Storage Diseases
This ClinicalTrials.gov registry entry describes ongoing recruitment for a registry of patients diagnosed with various lysosomal storage dis…
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CLINICAL_TRIALS research · 2026-03-30Clinical Trial Update: ISP-001: Sleeping Beauty Transposon-Engineered B Cells for MPS I [Phase 1]
Phase 1 clinical trial update for ISP-001, Immusoft's Sleeping Beauty transposon-engineered B cells intended to treat Mucopolysaccharidosis …
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CLINICAL_TRIALS research · 2026-03-17Clinical Trial Update: PEARL (PrEnAtal Enzyme Replacement Therapy for Lysosomal Storage Disorders) [Phase 1]
Phase 1 prenatal enzyme replacement therapy trial PEARL is currently recruiting for multiple lysosomal storage disorders including MPS I, MP…
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CLINICAL_TRIALS research · 2026-03-12Clinical Trial Update: RGX-111 Gene Therapy in Patients With MPS I [Phase 1, Phase 2]
The RGX-111 gene therapy trial for Mucopolysaccharidosis Type I is currently suspended in Phase 1/2.
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CLINICAL_TRIALS research · 2026-02-11Clinical Trial Update: MPS (RaDiCo Cohort) (RaDiCo-MPS)
An update on the RaDiCo-MPS clinical trial cohort sponsored by INSERM, recruiting patients with several mucopolysaccharidosis types I-IX and…
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CLINICAL_TRIALS research · 2026-02-09Clinical Trial Update: Longitudinal Study of Neurodegenerative Disorders
This clinical trial update describes a longitudinal study of several neurodegenerative and lysosomal storage disorders. The study is current…
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CLINICAL_TRIALS research · 2026-02-02Clinical Trial Update: Study to Evaluate the Safety and Efficacy of Adalimumab in MPS I, II, and VI [Phase 1, Phase 2]
A clinical trial is evaluating the safety and efficacy of adalimumab in mucopolysaccharidosis types I, II, and VI; the study is currently ac…
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CLINICAL_TRIALS research · 2026-01-26Clinical Trial Update: Cardiac Structure and Function in MPS
This clinical trial update shows ongoing recruitment for a study evaluating cardiac structure and function in patients with mucopolysacchari…
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CLINICAL_TRIALS research · 2025-10-30Clinical Trial Update: Evaluation of Intravenous Laronidase Pharmacokinetics Before and After Hematopoietic Cell Transplantation in Patients With Mucopolysaccharidosis Type IH.
This clinical trial update evaluates intravenous laronidase pharmacokinetics before and after hematopoietic cell transplantation in patients…
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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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EMA approval · 2024-10-17EMA Authorised: Aldurazyme (laronidase) — Sanofi B.V.
The EMA authorised Aldurazyme (laronidase) for mucopolysaccharidosis type I, with Sanofi B.V. as the marketing authorisation holder (decisio…
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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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FDA approval · 2024-07-10FDA Approved: NAGLAZYME — Supplemental Application (BioMarin Pharmaceutical Inc.)
FDA approves a supplemental application for NAGLAZYME (galsulfase) by BioMarin Pharmaceutical Inc.
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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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FDA approval · 2023-12-14FDA Approved: ALDURAZYME — Supplemental Application (Genzyme Corporation)
FDA approved a supplemental application for ALDURAZYME (Laronidase) submitted by Genzyme Corporation.
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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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FDA approval · 2019-12-04FDA Approved: ALDURAZYME — Supplemental Application (Genzyme Corporation)
FDA approved a supplemental application for ALDURAZYME (laronidase) submitted by Genzyme Corporation.
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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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FDA approval · 2013-04-03FDA Approved: ALDURAZYME — Supplemental Application (Genzyme Corporation)
FDA approved a supplemental application for Aldurazyme (laronidase) from Genzyme Corporation. This constitutes a supplemental approval of th…
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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…