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Clinicians have an important role in helping donation centers make the most of exciting advances and meet recent challenges.
The CRL is related to observations of a third-party manufacturing facility as part of a standard prelicense inspection.
Approval was based on data from the AGAVE-201 trial and is for use in adult and pediatric patients who weigh at least 40 kg.
Acalabrutinib was previously granted priority review in October 2024 based on results from the phase 3 ECHO trial.
In a study SCT appeared linked to an increased risk of pregnancy-associated VTE, pulmonary embolism, and isolated DVT.
Patients receiving mitapivat showed a statistically significant hemoglobin response rate compared with placebo.
Study data show the VAS-101 gel formulation helps preserve therapeutic efficacy and extend useful longevity of stored RBCs.
The tranexamic acid and placebo groups did not differ in occurrence of thrombotic events or veno-occlusive disease.
This novel JAK2 inhibitor features reduced off-target suppression of JAK1, JAK3, TYK2, and other kinases.
Questions surrounding informed consent, mandatory versus optional biopsy, and safety for adults and children are addressed.
In a phase 1 trial presented at ASH 2024, clinical benefit was observed in 100% of patients receiving LYT-200 monotherapy.
The new collaboration seeks to improve local PNH care in 53 countries whose health care systems have limited resources.
A review article describes current clinical knowledge of the role of inflammation and the activity of immune cells in SCD.
Juan Jose Rodriguez-Sevilla, MD, PhD, summarizes current clinical knowledge about inflammation within MDS pathobiology.
The results are from an ongoing international study of the two-agent combination in relapsed or refractory disease.
A study's efficacy and safety findings show promise for lovo-cel as a one-time intervention for SCD with history of stroke.
This approval for patients aged 12 years and older was based on findings from the explorer7 phase III clinical trial.
An MDS HSPC signature generated from the model presents possible targets for treatment in patients with low-risk disease.
The study's results especially highlight potential aberrant gene regulation by ZMAT2 and SMARCD3.
The study showed a mortality risk reduction benefit in certain mutations while for others larger studies are needed.
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