High-dose pharmaceutical grade biotin (MD1003) in amyotrophic lateral sclerosis: A pilot study.

ABSTRACT:

Background

Oligodendrocytes (OGs) provide metabolic support to motor neurons (MNs) and are implicated in the pathophysiology of amyotrophic lateral sclerosis (ALS). MD1003, or high-dose Pharmaceutical grade Biotin (hdPB), may improve disability in progressive multiple sclerosis patients via augmentation of OG or MN energy levels. Here, we assessed the safety and efficacy of MD1003 in ALS patients.

Methods

This single centre, randomised, double-blind, placebo-controlled trial included patients aged 25-80 years with probable or definite ALS. Patients were assigned (2:1), using a computer-generated randomisation list, to receive oral MD1003 (300 mg/day) or placebo treatment for 24 weeks. The primary outcome, safety, was analysed in all patients who received at least one dose of

SUBMITTER: Juntas-Morales R 

PROVIDER: S-EPMC7046518 | biostudies-literature | 2020 Feb

REPOSITORIES: biostudies-literature