A Retrospective, Natural History Study in Children With CLN2

首次发布于 2020 年 7 月 21 日

Cerliponase Alfa Observational Study in the US

计划入组 35 人 · 首次发布于 2020 年 7 月 20 日

A Phase 1/2 Open-Label Dose-Escalation Study to Evaluate Safety, Tolerability, Pharmacokinetics, and Efficacy of Intracerebroventricular BMN 190 in Patients With Late-Infantile Neuronal Ceroid Lipofuscinosis (CLN2) Disease

计划入组 24 人 · 首次发布于 2013 年 7 月 24 日

adeno-associated viral vector-based gene therapy (neuronal ceroid lipofuscinosis)

Cerliponase alfa(Brineura)

Poliovirus type 2 antigen (formaldehyde inactivated)(Ipol, Kinrix, Pediarix, Pentacel, Quadracel, Vaxelis)

Fractyl Health

MEREO BIOPHARMA 2 LIMITED

United Kingdom

UpJohn US 2 LLC

United States

Latus Bio Receives FDA IND Clearance for LTS-101 Gene Therapy to Treat Fatal CLN2 Disease in Children

FDA Expands Brineura Approval to Include All Ages with CLN2 Disease

FDA Grants Breakthrough Therapy Designation to Adrabetadex for Infantile-Onset Niemann-Pick Disease Type C