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Article

Stem cell transplantation for osteopetrosis in patients beyond the age of 5 years

18 Mar 2019

Abstract excerpt

Abstract Osteopetrosis (OP) is a rare disease caused by defective osteoclast differentiation or function. Hematopoietic stem cell transplantation (HSCT) is the only curative treatment available in the infantile “malignant” form of OP. Improved clinical and genetic diagnosis of OP has seen the emergence of a cohort of patients with less severe and heterogeneous clinical presentations. This intermediate form of OP...

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PMID: 30885997
DOI: 10.1182/bloodadvances.2018025890

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