The primary mission for the unit is to ensure that Danish children and adolescents with rare diseases gain access to the newest medicine available. In collaboration with pharmaceutical companies, we systematically test the efficacy, efficiency and safety of new medicine used to treat a variety of skeletal dysplasias, rare metabolic, lysosomal, and neurological disorders, as well as rare heart and lung diseases. Long term, these trials help secure approval of new medicines, but they also make it possible to offer new, innovative, and life-saving treatments to children who would otherwise go without treatment.

Our aim as a unit is to be able to undertake all relevant opportunities for clinical trials testing new treatments, which can ensure that children and young people with rare diseases receive the treatments and care that they need and deserve.

​​​Researchers

• Merete Ljungberg, M.D., Center for Rare Diseases
• Hanne Hove, M.D., DMSc, Head of Craniofacial team and team for Skeletal Dysplasia, Center for Rare Diseases
• Allan Lund, Professor, M.D, DMSc, Head, Center for Inherited Metabolic Diseases
• Line Aagaard Nolting, M.D., Center for Rare Diseases
• Malene Landbo Børresen, M.D., Ph.D., Department of Pediatric Neurology
• Alfred Peter Born, M.D., Ph.D., Department of Pediatric Neurology
• Christina Høi-Hansen, Professor, DMSc, Department of Clinical Medicine, Copenhagen University & Department of Pediatric Neurology
• Kim Gjerum Nielsen, Professor, MD, DMSc, Chief Consultant, Danish PCD & chILD Centre, CF Centre Copenhagen, Paediatric Pulmonary Service, Department of Paediatrics and Adolescent Medicine ​

Funding

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