Article

Hematopoietic stem cell transplantation in a patient with proteasome-associated autoinflammatory syndrome (PRAAS)

2021-08-17

Abstract excerpt

Background Proteasome-associated autoinflammatory syndromes (PRAASs) form a family of recently described rare autosomal recessive disorders of disturbed proteasome assembly and proteolytic activity caused by mutations in genes coding for proteasome subunits. The treatment options for these proteasome disorders consist of lifelong immunosuppressive drugs or Janus kinase inhibitors, which may have partial efficacy a...

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Literature Corpus work: e1bdaf88-57eb-549f-a858-16bb39abe58c
DOI: 10.1016/j.jaci.2021.07.039

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