February 23, 2024
A study published in Haemophilia looked at how common sarcopenia (loss of muscle mass and strength) was in men diagnosed with severe hemophilia A who had recurrent joint bleeds, compared to healthy men. A modified Sonographic Thigh Adjustment Ratio was used as a measure of muscle mass.
February 7, 2024
Over seven years of follow-up, a single dose of Roctavian (valoctocogene roxaparvovec-rvox) reduced the yearly bleeding rate in adults with severe hemophilia A by up to 96%. According to an update on a Phase 1/2 clinical trail in the UK that is testing the safety and efficacy of the gene therapy at various doses, no new safety issues were found.
January 31, 2024
The National Bleeding Disorders Foundation (NBDF) Board of Directors announced that Phil Gattone, M.Ed will be their next president and chief executive officer. Gattone was president and CEO of the Epilepsy Foundation of America from 2012-2019 and is succeeding Len Valentino, MD, who was president and CEO of the NBDF since 2020.
January 17, 2024
After almost three years of treatment with Jivi (damoctocog alfa pegol), a multinational group of hemophilia A patients showed a reduced annual bleeding rate. A study, looking at real world effectiveness of the treatment, showed there were no reported side effects or discontinuations.
January 12, 2024
The first hemophilia A gene therapy infusion to be delivered outside a clinical trial setting was recently given to a patient in California. The patient, who has severe hemophilia A, received a single infusion of Roctavian (BioMarin) at CIBD in Orange, CA.
December 22, 2023
In a phase 1/2a clinical trial by Centessa Therapeutics, SerpinPC, a subcutaneous injection given every two weeks, showed the treatment to be safe and that bleeds of patients were reduced to almost zero in adults with hemophilia A or B. The patients in the study were in their third year of treatment and had their annualized bleeding rate drop by 96%.
Dec 20, 2023
Data from the Phase 3 HOPE-B trial shared at the 65th American Society of Hematology (ASH) Annual meeting shows bleeding rates and the use of replacement therapies among men with severe or moderately severe hemophilia B remain low at least three years after a single dose of the approved gene therapy Hemgenix (etranacogene dezaparvovec).
December 6, 2023
A first patient has been dosed in a Phase 1/2a clinical trial that is assessing safety and efficacy of ASC Therapeutics' ASC618, a second-generation gene therapy for hemophilia A. The trial aims to enroll about 12 men with severe or moderately severe hemophilia A and the treatment is expected to be "more durable and affordable."
November 29, 2023
The US Food and Drug Administration has granted Orphan Drug Designation and Rare Pediatric Disease Designation for an investigational hemophilia A therapy being developed by Sernova. The therapy would use their proprietary Cell Pouch technology that targets a patient's blood outgrowth endothelial cells.
November 11, 2023
Genevant Sciences is teaming up with Novo Nordisk to develop a gene-editing treatment for hemophilia A. The gene editing is used to correct, add, or delete a DNA sequence for the bleeding disorder.
November 6, 2023
A study published in The Lancet Haematology examined the use of tranexamic acid (TA) vs. recombinant von Willebrand factor replacement therapy (rVWF). The small study found that scores were better after cycles with TA than with rVWF, but neither therapies corrected bleeding scores to a normal range.
October 13, 2023
In a global survey, more than half of patients report having only fair or poor health, but tell their healthcare provider that they are fine. The survey noted that more than 50% try to hide their symptoms from others about once a week or more often. Most were concerned with being treated differently, according to the survey's findings.
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