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hemophilia and kids | Image credit: Denira - stock.adobe.com
FDA Approves Jivi for Pediatric Patients Aged 7 to 12 Years With Hemophilia A

May 19th 2025

The FDA previously approved Jivi in 2018 to treat adults aged 12 years and older with hemophilia A.

FDA approval-Olivier Le Moal-stock.adobe.com
FDA Approves Fitusiran for Reducing Bleeds in Patients With Hemophilia A/B

March 28th 2025

hemophilia-jarun011-stock.adobe.com
Research Supports Need for Diversity in Hemophilia Trials and Reducing Resource Strain

December 24th 2024

FDA-Deni-stock.adobe.com
First Subcutaneous Prophylaxis for Hemophilia A and B With Inhibitors Approved by FDA

December 23rd 2024

Top 5 Rare Blood
Top 5 Most-Read Articles on Rare Blood Disorders in 2024

December 20th 2024

Treating PNH: Complement Inhibitors, Administration Modes, and Coordinated Care Needs

Join experts as they explore PNH pathophysiology, treatment strategies, clinical trials, and management challenges. Gain essential insights to enhance patient outcomes and optimize PNH treatment.

View the latest AJMC Peer Exchange on paroxysmal nocturnal hemoglobinuria (PNH)

More News

sickle cell graphic
May 12th 2022

Adolescent, Young Adult Males With Sickle Cell Disease Lack Knowledge of Fertility Implications, Study Shows

Rose McNulty
Improved management of sickle cell disease has vastly improved life expectancy and reduced morbidity for affected patients, but the disease itself and its treatments carry fertility risks that many young patients and their caregivers are unaware of.
doctor with tablet as various symbols related to healthcare pass by
October 20th 2021

Cancer Treatments, Orphan Drugs, Gene Therapies Represent Big Areas to Watch in Specialty Drug Pipeline

Skylar Jeremias
During her presentation at the Academy of Managed Care Pharmacy Nexus 2021 meeting, Aimee Tharaldson, PhD, highlighted the most anticipated specialty pharmacy drugs awaiting FDA approval, including several treatments for cancer and rare diseases.
Working on gene therapy
April 23rd 2021

Gene Transfer Therapy Requires Further Investigation Despite Potential Approval, Review Says

Larry Hanover
After a more than 35-year pursuit, the licensing of a gene therapy product for treating hemophilia may be available within 2 years.
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