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A Phase 1/2 Open-Label Dose-Escalation Study to Evaluate the Safety, Tolerability, Pharmacokinetics and Efficacy of Intracerebroventricular BMN 250 in Patients with Mucopolysaccharidosis Type IIIB (MPS IIIB, Sanfilippo Syndrome Type B) (BMN250-201) https://clinicaltrials.gov/ct2/show/NCT02754076?term=BMN250draw=2rank=2

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Okur İ. (Executive), Börcek A. Ö., Ezgü F. S., Tümer L., İnci A., Soysal Acar A. Ş., et al.

Project Supported by Private Organizations in Other Countries, 2017 - 2020

  • Project Type: Project Supported by Private Organizations in Other Countries
  • Begin Date: October 2017
  • End Date: September 2020