Rare disease business of AstraZeneca, based at Boston in Massachusetts and formed from Alexion Pharmaceuticals, which was founded in 1992 at New Haven in Connecticut and acquired by AstraZeneca in 2021. Develops and markets medicines for rare and devastating diseases, including the complement inhibitors eculizumab and ravulizumab for paroxysmal nocturnal haemoglobinuria, atypical haemolytic uraemic syndrome and myasthenia gravis, enzyme replacement therapies for hypophosphatasia and lysosomal acid lipase deficiency and a pipeline in complement biology, neurology and metabolic disease.
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