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uniQure NV is a gene therapy company. It develops treatments and platforms for patients suffering from genetic and other devastating diseases. Its products and services are focused on hemophilia, Huntington's disease, and cardiovascular diseases. The company is focused on the development of the pipeline of gene therapies with the collaboration of Bristol Myers Squibb for cardiovascular diseases.
Pharming Group is a biopharmaceutical company focused in transforming the lives of patients with rare, debilitating, and life-threatening diseases. The company is actively developing and commercializing a diverse portfolio of innovative treatments, including both biologics and small molecule therapies. Its commercial products are RUCONEST, approved for the treatment of acute attacks in adult and adolescent patients with hereditary angioedema, or HAE; and Joenja (leniolisib), a small-molecule kinase inhibitor, developed for the treatment of activated phosphoinositide 3-kinase delta syndrome.