Saves the questions and what to expect into your notes, next to the visit they belong to.
Keep this study
Lead
Beam Therapeutics Inc.
Age
12–35
Sex
ALL
Healthy volunteers
Not accepted
You may be eligible if
1. Age ≥12 years to ≤35 years
2. Documented diagnosis of sickle cell disease with βS/βS, βS/β0, or βS/β+ genotypes.
3. Severe SCD defined by the occurrence of at least 4 severe VOCs in the 24 months prior to screening despite receiving hydroxyurea or other supportive care measures
You may not be if
1. HbF levels \>20%, obtained at the time of screening on or off hydroxyurea therapy
2. Previous receipt of an autologous or allogeneic HSCT or solid organ transplantation
3. Available and willing matched sibling donor
4. Definitive diagnosis of moyamoya syndrome based on screening brain MRA
