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

An optimal approach to rare disease management based on national experiences in Europe, North America and East Asia

Michigan to destroy leftover blood samples it took from newborns

France: Official number of cases per rare disease

International Neonatal Screening Day website now available

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