Useful resources
Resources on this website:
- Tips on safe data sharing; Are you protecting your personal data and being a ‘smart’ research participant?
- Audentes’ Letter to the Patient Community (31 January 2019)
- Frequently Asked Questions (and answers) from Audentes about AT132; their gene therapy product for x-linked myotubular myopathy (8 April 2017)
Resources on other websites:
- Understanding the drug discovery process (from the website Compound Interest)
- Basic overview of the FDA (USA) drug approval process (from the MTM-CNM Family Connection)
- MTM & CNM Factsheet (from Muscular Dystrophy UK)
Patient organisations and support groups:
- Joshua Frase Foundation (USA)
- MDUK (UK)
- MTM-CNM Family Connection (USA)
- Myotubular Myopathy Resource Group (USA)
- Myotubular Trust (UK)
- Where There’s A Will There’s A Cure (USA)
- ZNM – Zusammen Stark! (Germany)
Registry materials: