DMD Guide for Families
Latest!
New 2018 DMD Guide for Families now available in US & UK.
Post marketing surveillance
Collection of safety and efficacy data once a drug has received conditional marketing approval
Publications
Latest publications!
Care guidelines for DMD & SMA - significant updates published
Industry
Download our industry brochure
Trial sites and patients, advisory support, study support, training
Disease information
Myotubular and centronuclear myopathy disease section now live!
IRDiRC recommends...
IRDiRC recommends our Patient Registries and also our Animal model SOPs
TACT
TREAT-NMD Advisory Committee for Therapeutics - application & review process, past reviews, committee etc
BioBanks
A unique network of biobanks storing quality DNA, cell & tissue samples on rare diseases and NMDs
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By supporting the work of the TREAT-NMD Alliance you can help us advance research
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- Chair's Review of 2018 As this is the last Newsletter of 2018, it gives us a good opportunity ...
- Rare Disease Day 2019 theme announced In 2019, the theme will be "Bridging health and social care". This special day...
- TREAT-NMD Masterclass in Spinal Muscular Atrophy TREAT-NMD hosted its inaugural two day masterclass in spinal muscular atrophy (SMA) on the 19...
- See Latest Newsletter
- Rare Disease Day 2019
28 Feb 2019 Rare Disease Day takes place on the last day of... - Orphan Drug Congress
17 -18 Jun 2019 With the accomplishment of preceding Rare Diseases Congress in 2018,... - 2019 National Limb Girdle Muscular Dystrophy Conference
30 Aug 2019 - 2 Sep 2019 This is the first national conference dedicated to all forms... - See All Events