Introduction Duchenne Muscular Dystrophy (DMD) is a severe genetic disorder characterized by progressive muscle degeneration and weakness. In March 2024, the U.S. Food and Drug Administration (FDA) approved Duvyzat (givinostat), marking a significant advancement in…
Hematology is the branch of medicine that focuses on blood, bone marrow, and related disorders — from anemia to leukemia. In recent years, research and innovation have led to extraordinary progress, offering new hope for…
Diffuse large B-cell lymphoma (DLBCL) is the most common type of non-Hodgkin lymphoma (NHL), accounting for about 30-40% of all NHL cases. It is an aggressive but potentially curable cancer, with treatment advancements improving survival rates.…
🔍What is Chronic Lymphocytic Leukemia? Chronic Lymphocytic Leukemia (CLL) is a slow-growing blood cancer characterized by the clonal proliferation of mature B‑lymphocytes in bone marrow, blood, and lymphoid tissues. It primarily affects older adults, with…
🧬 What is the Hemophilia Type A? Hemophilia A, or classic hemophilia, is a rare genetic bleeding disorder caused by a deficiency or dysfunction of Factor VIII (FVIII), a crucial blood-clotting protein. It is an…
Multiple myeloma (MM) is a type of blood cancer that affects plasma cells, a crucial part of the immune system responsible for producing antibodies. When these cells become cancerous, they multiply uncontrollably in the bone…
HYMPAVZI is the first and only anti-tissue factor pathway inhibitor (anti-TFPI) approved in the U.S. for the treatment of hemophilia A or B and the first hemophilia medicine approved in the U.S. to be administered…
Phil GattonePresident and CEO, National Bleeding Disorders Foundation“…. Qfitlia takes a novel approach to providing protection for people living with hemophilia while reducing the frequency of dosing for patients and their families.” Hemophilia is a…
In January 2025, the FDA approved Grafapex™ (treosulfan) in combination with fludarabine as a conditioning regimen for allogeneic hematopoietic stem cell transplantation (alloHSCT). This approval applies to adult and pediatric patients aged one year and…
NEW YORK, April 8, 2025 (GLOBE NEWSWIRE) — TG Therapeutics, Inc.(TGTX) announced today that it will publish data on BRIUMVI® (ublituximab-xiiy) for the treatment of patients with relapsing multiple sclerosis (RMS). Michael S. Weiss, Chief…
In a significant advancement for hemophilia care, the U.S. Food and Drug Administration (FDA) has approved Alhemo® (concizumab-mtci) as a once-daily prophylactic treatment for adults and children aged 12 years and older with hemophilia A…
On 15 November 2024, the US Food and Drug Administration (FDA) approved a new drug called Revuforj (revumenib) for the treatment of adult and paediatric patients aged 1 year and older with relapsed or refractory…
