Records containing: MPS I
This is the wording recorded or indexed by the source. An agency index term is not necessarily an authorised indication. Co-listed drug names are not linked to this term as a treatment relationship.
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CLINICAL_TRIALS study record update · 2026-03-17PEARL (PrEnAtal Enzyme Replacement Therapy for Lysosomal Storage Disorders)
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CLINICAL_TRIALS study record update · 2026-02-09Longitudinal Study of Neurodegenerative Disorders
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CLINICAL_TRIALS study record update · 2026-01-26Cardiovascular Structure and Function in Mucopolysaccharidosis Types I and IVA
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CLINICAL_TRIALS study record update · 2023-04-11A Phase 1/2 Open-Label, Sequential Dose-Escalation, Safety, Tolerability and Efficacy Study of SIG-005 in Adult Patients With Mucopolysaccharidoses 1 (MPS-1)
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CLINICAL_TRIALS study record update · 2023-01-26A Phase I / 2, Multicenter, Open-label, Single-dose, Dose-ranging Study to Assess the Safety and Tolerability of SB-318, a rAAV2/6-based Gene Transfer in Subjects With Mucopolysaccharidosis I (MPS I)
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CLINICAL_TRIALS study record update · 2022-03-17The Intensively Follow-up Examinations for Asymptomatic MPS I Infants From Nationwide Newborn Screening Program for Mucopolysaccharidoses in Taiwan.
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CLINICAL_TRIALS study record update · 2018-09-27MT2011-21C Laronidase (Aldurazyme TM) Enzyme Replacement Therapy (ERT) With Hematopoietic Stem Cell Transplantation (HSCT) for Hurler Syndrome (MPS IH).
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CLINICAL_TRIALS study record update · 2018-01-24Ultrasound Findings of Finger, Wrist and Knee Joints in Mucopolysaccharidosis