Mucopolysaccharidosis NFZ — 19 facilities in 19 cities

Mucopolysaccharidoses are a group of rare lysosomal diseases where enzyme deficiencies cause glycosaminoglycan accumulation. They manifest with skeletal deformities, liver and spleen enlargement, respiratory impairment, and vision or hearing decline. NFZ covers enzymatic diagnostics, enzyme replacement therapy for specific types (II, IVA, VI, VII), and multidisciplinary care at referral centers.