Mucopolysaccharidosis type 1

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Information
Disease name
Mucopolysaccharidosis type 1
Disease ID
Description
Disease area statistics
[No Data.]
Chromosome band
[No Data.]
Annotation
Genes Mutation Description Source Links
NCT ID Status Phase Summary Start date Completion date
NCT06406153 Completed Phase 3 Efficacy and Safety of P046 (Laronidase-CinnaGen) Compared to Aldurazyme® in MPS I Patients September 10, 2022 November 18, 2023
NCT03655223 Enrolling by invitation Early Check: Expanded Screening in Newborns October 15, 2018 December 31, 2025
OrphaNumber from OrphaNet (Orphanet)
579