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JR-141 long-term extension study in mucopolysaccharidosis type II
2022-503142-41-00Phase 3Authorised
- Sponsor
- JCR Pharmaceuticals
- Start
- May 2024
- 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, open-label trial of pabinafusp alfa in mucopolysaccharidosis type 2 (2 cohorts), sponsored by JCR Pharmaceuticals. Primary endpoints: Cerebrospinal fluid biomarkers and opening pressure, Neuropsychological assessment and 1 more. Planned start May 2024; status: authorised.
02
Drugs
pabinafusp alfa
03
Conditions
mucopolysaccharidosis type 2
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