Diseases [C] » Hemic and Lymphatic Diseases [C15] » Hematologic Diseases » Bone Marrow Diseases » Bone Marrow Failure Disorders » Congenital Bone Marrow Failure Syndromes
Diseases [C] » Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16] » Infant, Newborn, Diseases » Congenital Bone Marrow Failure Syndromes
Description
Inherited syndromes characterized by deficiency or absence of various blood cells due to mutations that affect HEMATOPOIETIC STEM CELLS development and proliferation. MeSH
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Subtype Terms (3)
Anemia, Hypoplastic, Congenital
1 experimental drug
Dyskeratosis Congenita
12 drugs (11 approved, 1 experimental)
Shwachman-Diamond Syndrome
16 drugs (12 approved, 4 experimental)
Phase 2 Indicated Drugs (5)
Phase 1 Indicated Drugs (2)
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UMLS Data
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