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Hemophilia A

Current Clinical Trials: Hemophilia A

Study: Phase I/II Gene Therapy Study of BMN 270 for Hemophilia A

Indication: Hemophilia A

Investigational Therapeutic: BMN 270

Study Type: Phase 1/2

Goal: A Phase 1/2, Dose-Escalation Safety, Tolerability and Efficacy Study of BMN 270, an Adenovirus-Associated Virus Vector–Mediated Gene Transfer of Human Factor VIII in Patients with Severe Haemophilia A

Status: Ongoing

Additional information: View on EU Clinical Trials Registry and clinicaltrials.gov.

BioMarin

Diseases

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

Products

Vimizim®
Kuvan®
Naglazyme®
Aldurazyme®
Firdapse®

Patient/Physician Support Contact Information

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

Global Medical Information

Contact Global Medical Information

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