Interestingly a drug, sotatercept, an activin receptor type IIA ligand trap, acts by inhibiting signaling downstream of activin and other TGFβ superfamily members is currently being tested in patients with DBA (ClinicalTrials.gov https://clinicaltrials.gov/ct2/show/NCT01464164) [51]. Here, TGFB1 is linked to Diamond-Blackfan anemia.