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Phase III study of JR-141 in mucopolysaccharidosis type II
2024-512289-33-00Phase 3Authorised
- Sponsor
- JCR Pharmaceuticals
- Start
- Aug 2022
- Modality
- Replacement Enzymes → Therapeutic Enzymes → Recombinant Proteins and Enzymes, Fc-Fusion Proteins → Carrier/Scaffold Proteins → Recombinant Proteins and Enzymes
- Countries
- 12
01
Overview
Phase 3, randomised, open-label trial of pabinafusp alfa in mucopolysaccharidosis type 2 (4 cohorts), sponsored by JCR Pharmaceuticals. Primary endpoints: Change in BSID-III cognitive raw score and Change in cerebrospinal fluid heparan sulfate. Planned start Aug 2022; status: authorised.
02
Drugs
pabinafusp alfa
03
Conditions
mucopolysaccharidosis type 2
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