Adults and children affected by myotubular and centronuclear myopathies, their families, doctors and carers attended a family conference jointly hosted by the Myotubular Trust and ZNM – Zusammen Stark in London, on 14 and 15 July 2018.
Highlights included research updates on gene therapy clinical trials, upcoming clinical trials using DNM2 down regulation, drug repurposing for x-linked myotubular myopathy, day to day care, tracheostomy management, latest best practice in respiratory care, genes and genetic counselling.
