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Mucopolysaccharidosis IH/S
Clinical trial pipeline · Data from ClinicalTrials.gov
See which Mucopolysaccharidosis IH/S trials you may qualify forClinical trial pipeline · Data from ClinicalTrials.gov
See which Mucopolysaccharidosis IH/S trials you may qualify forRGX-111 is a gene therapy which is intended to deliver a functional copy of the α-L-iduronidase (IDUA) gene to the central nervous system. This is a safety and…
The Mucopolysaccharidosis I (MPS I) Registry is an ongoing, observational database that tracks the outcomes of patients with MPS I. The data collected by the MP…
Long-term follow-up of subjects who received SB-318, SB-913, or SB-FIX in a previous trial and completed at least 52 weeks post-infusion follow-up in their prim…