Hematology

The approval makes etuvetidigene autotemcel (Waskyra) the first cell-based gene therapy for the treatment of Wiskott-Aldrich syndrome.

Morris discusses this latest trial in a long list of phase 3 studies which failed to evaluate arginine therapy’s value in vaso-occlusive pain episodes in SCD.

Al-Samkari discusses the positive results from the VAYHIT2 trial, which investigated ianalumab in combination with eltrombopag in adults with ITP.

Gwarzo discussed the shortcomings faced by many patients with SCD during pain crises, due to less frequent opioid prescription than guidelines recommend.

Announced by Cogent Biosciences, these data indicate bezuclastinib’s capacity for improving patient-reported symptoms of mast cell burden.

The approval of Ayrmid’s omidubicel-onlv makes it the first hematopoietic stem cell transplant therapy to treat patients with severe aplastic anemia.

Strunk highlights reduced length of stay and superior outpatient pain management due to palliative care in hospitalized patients.

Rusfertide shows promising results as a first-in-class therapy for polycythemia vera, demonstrating sustained hematocrit control and safety in recent studies.

GLP-1 RA use was linked to a significant survival benefit and reduced risk of iron deficiency anemia in patients with celiac disease.

Results from the phase 3 trial show consistent reductions in annualized bleeding rate after 12 months of once-weekly treatment.

A study reveals that APOE e4 carriers with atrial fibrillation on Eliquis face increased intracranial hemorrhage risk, highlighting critical genetic implications.

Explore key FDA decisions in Q4 2025, impacting treatments for atopic dermatitis, chronic rhinosinusitis, and rare diseases.

Denecimig may offer a prophylaxis bleed treatment option for a range of patients across dosing frequencies, age groups, and condition severity.

This Sickle Cell Awareness Month, HCPLive draws attention to recent advancements made in SCD treatment and how much still needs to be done to address it.

An audio recap of the top 5 stories in healthcare news from the week of 08/31-9/5.

Stay updated with the latest healthcare breakthroughs, including FDA news and phase 3 clinical trial data, in this week's essential news roundup.

FDA expands Vonvendi approval for all von Willebrand disease types, enhancing treatment options for adults and children with this rare condition.

Agios announces a 3-month extension for mitapivat's FDA review.

An audio recap of the top 5 stories in healthcare news from the week of 08/24-08/30.

Stay updated with the latest healthcare breakthroughs, including FDA news, vaccine guidelines, and orforglipron data, in this week's essential news roundup.

If approved, the investigative therapy could provide a frontline treatment for patients with SAA who do not have matched sibling donors for cell transplantation.

Stay updated with the latest healthcare breakthroughs, including FDA news and new guidelines, in this week's essential news roundup.

This new monoclonal antibody has achieved a statistically significant improvement in time to treatment failure, indicating a potential to revolutionize ITP.

The FDA approved generic versions of Venofer (iron sucrose) injection from Viatris Inc and Amphastar Pharmaceuticals.

This FDA News Month in Review provides a round-up of regulatory decisions from July 2025.