| Disease Subtype(s) |
HCT Disease Inserts Required |
Cellular Therapy Disease Inserts Required |
- Diamond-Blackfan anemia (pure red cell aplasia) (312)
- Telomere biology disorders including dyskeratosis congenita (DKC1, TERT, TERC, and other mutations) (307)
|
Forms 2028 & 2128 |
No disease inserts required |
|
Forms 2029 & 2129 |
- Severe congenital neutropenia (Elastase deficiency / ELANE or Kostmann disease / HAX1 mutations) (460)
|
No disease inserts required |
- Shwachman-Diamond syndrome (DNAJC21, EFL1, or SBDS mutations) (305)
- Germline SAMD9 variant (MIRAGE Syndrome) (2311)
- Germline SAMD9L variant (SAMD9L-related Ataxia Pancytopenia Syndrome) (2312)
- Other inherited bone marrow failure syndrome (339)
|
Forms 2028 & 2128 |
Last modified:
May 29, 2024
