A Mortal Complication in a Case with Mucopolysaccharidosis type I Following Hematopoietic Stem Cell Transplantation: Pulmonary Haemorrhage

• Main Authors: Havva Yazıcı (Author), Ebru Canda (Author), Esra Er (Author), Baris Malbora (Author), Burcu Ozturk Hismi (Author), Huseyin Onay (Author), Serap Aksoylar (Author), Sema Kalkan Uçar (Author), Ferda Ozkinay (Author), Mahmut Çoker (Author)
• Format: Book
• Published: Galenos Publishing House, 2021-08-01T00:00:00Z.
• Subjects: article
• Online Access: Connect to this object online.

Mucopolysaccharidosis type I (MPS I) is a lysosomal storage disease due to mutations within the gene IDUA encoding the 'α-L-iduronidase'. The clinical manifestations concern multisystemic involvement. There are two disease modifying therapies, enzyme replacement therapy and haematopoietic...