Longitudinal Assessment of Atypical Tripeptidyl Peptidase 1 Enzyme Deficiency Patients

5 participants · First posted Sep 23, 2019

A Safety, Tolerability, and Efficacy Study of BMN 190 in Pediatric Patients < 18 Years of Age With CLN2 Disease

14 participants · First posted Feb 10, 2016

A Multicenter, Multi-national Open-label Program to Provide BMN 190 to Patients Diagnosed With CLN2 Disease

First posted Nov 15, 2016

tripeptidyl peptidase 1 stimulator (Weill Medical College of Cornell University)

Cerliponase alfa(Brineura)

USP30 inhibitors (Progenra)

NHI-1

United States

Xi'an No.1 Hospital

China

ALPHA-1 FOUNDATION, INC.

United States

FDA Expands Brineura Approval to Include All Ages with CLN2 Disease

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

Novel AAV Capsid Shows Unprecedented Brain Transduction Potential for Gene Therapy