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Congenital Bone Marrow Failure Syndromes
Inherited syndromes characterized by deficiency or absence of various blood cells due to mutations that affect HEMATOPOIETIC STEM CELLS development and proliferation.
Subtype terms (3)
More specific conditions that are subtypes of Congenital Bone Marrow Failure Syndromes in the MeSH hierarchy.
- Anemia, Hypoplastic, Congenital
1 drug experimental
- Dyskeratosis Congenita
21 drugs (17 approved, 4 experimental)
- Shwachman-Diamond Syndrome
16 drugs (13 approved, 3 experimental)
Drugs by status
Phase 2 trials (7)
Phase 1 trials (3)