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Cerliponase alfa

Current Clinical Trials: CLN2 Disease

 
Study: 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

Indication: CLN2 Disease (Late-Infantile Neuronal Ceroid Lipofuscinosis Type 2)

Investigational Therapeutic: BMN 190

Study Type: Phase 1/2

Goal: The purpose of this study is to determine whether BMN 190 is safe and effective in the treatment of patients with Late-Infantile Neuronal Ceroid Lipofuscinosis Type 2 (CLN2) disease.

Status: This study is ongoing, but not recruiting participants

Additional information: View on Clinicaltrials.gov

Study: A Phase 2 Open-Label Study to Evaluate Safety, Tolerability, and Efficacy of Intracerebroventricular BMN 190 in Patients With CLN2 Disease

Study Type: Phase 2

Goal: The purpose of this study is to evaluate the safety, tolerability, and efficacy of BMN 190 in patients with CLN2. The study is designed to assess disease progression in siblings of children enrolled in the 190-201 study.

Status: Enrolling participants by invitation only

Additional information: View on Clinicaltrials.gov

Cerliponase Alfa for CLN2 Disease

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BioMarin

Diseases

Lysosomal Storage Disorders (MPS I, MPS IVA, MPS VI) PKU, LEMS

Products

Vimizim®
Kuvan®
Naglazyme®
Aldurazyme®
Firdapse®

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Tel: 866.906.6100
Fax: 888.863.3361
E-mail: support@biomarin-rareconnections.com

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