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)
Phase of Trial: Phase I/II
Latest Information Update: 07 Feb 2018
At a glance
- Drugs BMN 250 (Primary)
- Indications Mucopolysaccharidosis III
- Focus Adverse reactions; First in man; Therapeutic Use
- Acronyms MPS IIIB
- Sponsors BioMarin Pharmaceutical
- 07 Feb 2018 Interim results were presented in a BioMarin Pharmaceutical media release.
- 07 Feb 2018 According to a BioMarin Pharmaceutical media release, interim data from this trial was presented at WORLDSymposium 2018.
- 05 Feb 2018 According to a BioMarin Pharmaceutical media release, data will be presented at the 14th Annual WORLDSymposiumTM 2018.
Most Recent Events
Table of Contents
- At a glance
- Trial Overview
- Trial Details
- Trial Centres
- Trial History