Experts and patient representatives discussed newborn screening registries, case definitions, and a roadmap for an equitable NBS system.
Metabern & ISNS meeting – Moving towards NBS as a system: Setting out the roadmap Read More »
A webinar on 9 Nov will explore drug development and collaboration with Rare Disease networks to improve patient care.
Drug development and how best to work with specialised Rare Disease networks – webinar Read More »
c4c launches a European campaign to raise awareness of paediatric clinical trials. Take the survey and share your opinion.
c4c – paediatric clinical trials awareness campaign Read More »
A free online seminar for paediatricians on early DMD diagnosis will launch in Spring 2022. Take the survey and register interest.
TREAT-NMD DMD Early Diagnosis Training Seminar Read More »
A collaborative network of European cystinosis support groups working to improve care and research for patients since 2016.
Cystinosis European Network Read More »
A comprehensive overview of CDG patient advocacy groups and representatives working to advance awareness, research and treatment options.
CDG Patient Groups and Advocates Read More »
An international network uniting professionals and patient associations to advance research in Congenital Disorders of Glycosylation.
A worldwide alliance of non-profit organizations providing support and advancing research for Niemann-Pick Disease communities.
Inpda: International Niemann–Pick Disease Alliance Read More »
An Australian network dedicated to supporting patients with Classical Homocystinuria while advancing research toward effective treatments.
HCU Network Australia Read More »
A parent-founded European organization uniting 41 national PKU associations to advance care and quality of life for PKU communities.
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