Queen Square Centre for Neuromuscular Diseases


Becker Muscular Dystrophy - Natural history to predict efficacy of exon skipping

Sponsor / Funder CINRG
CI Professor Kate Bushby
Sites Newcastle
Contact details becky.davis@ncl.ac.uk
More information

This is a multi-centre natural history study. It will be conducted at Cooperative International Neuromuscular Research Group (CINRG) sites and affiliated centres.

After a baseline evaluation, participants will have three follow-up visits over three years.

We will characterize the BMD phenotype and link specific abnormal dystrophin proteins with the range of clinical outcomes.