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Regulation (EC) № 141/2000 for orphan medicinal products – 20 years later

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Hereditary retinal dystrophies

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The Importance of Reference Centers and Registries for Rare Diseases: The Example of Alpha-1 Antitrypsin Deficiency

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European Reference Networks – Animation clip for patients and health professionals

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Regulation (EC) № 141/2000 for orphan medicinal products – 20 years later

by informer

Hereditary retinal dystrophies

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The Importance of Reference Centers and Registries for Rare Diseases: The Example of Alpha-1 Antitrypsin Deficiency

by informer

European Reference Networks – Animation clip for patients and health professionals

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Challenges for patients with congenital adrenal hyperplasia and their doctors and parents

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Generation of FX -/- and Gmds -/- CHOZN host cell lines for the production of afucosylated therapeutic antibodies

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Natural history of multiple sulfatase deficiency: Retrospective phenotyping and functional variant analysis to characterize an ultra-rare disease

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Standardized Data Structures in Rare Diseases: CDISC User Guides for Duchenne Muscular Dystrophy and Huntington’s Disease

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Оnline event of World Alliance of Pituitary Organizations (WAPO)

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Clinical, electrophysiological and genetic characteristics of childhood hereditary polyneuropathies

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Current treatment of sarcoidosis

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Survey on the XI National Conference for Rare Diseases and Orphan Drugs

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Osteogenesis imperfecta: towards an individualised interdisciplinary care strategy

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Short Bowel Syndrome: A Paradigm for Intestinal Adaptation to Nutrition?

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