Glutaryl-CoA dehydrogenase deficiency : a study showed that newborn screening programs for Glutaric aciduria type 1 have an overall positive effect on the neurological outcome but their success depends on the quality of therapy

Report NGO Committee for Rare Diseases on Rare Disease Day event “Rare Diseases: A Global Priority for Equity”

Campaign for the adoption of a Rare Disease strategy in Canada

Launch of the European Health Data Space

Mothers’ knowledge and attitudes about newborn screening in Jordan

MetabERN: Article on the Screen4rare initiative for newborn screening programmes in Europe

MetabERN: Study on the evolution of rare disease registries