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Bleeding and Clotting Disorder News & Archives

Emicizumab May Yield Improvements in Joint Health in Hemophilia Patients

Results presented at the American Society of Hematology (ASH) showed that emicizumab may yield clinically relevant improvements in joint health in patients with hemophilia. The results indicate that emicizumab has a favorable safety profile and has led to reductions in joint bleeds. See full article at

UniQure and Pfizer Share Updates on Gene Therapy Trials for Hemophilia B

Both UniQure and Pfizer shared updates on their gene therapy trials for hemophilia B patients at the annual American Society of Hematology (ASH) national meeting in Orlando, Florida. Both trials have been ongoing for more than a year and both show promise. See full article at

Takeda and Enzyre Partner to Develop Home Blood Test for Hemophilia Patients

Enzyre is teaming up with Takeda to develop a diagnostic device that will allow people with hemophilia to determine their blood coagulation status at home. The goal is to create a device that enables patients to determine their status in nearly real time and share results with treating physicians through a mobile app. See full article at

Study Examines Adherence Rates for Hemophilia Patients Using Emicizumab

A new study shared at the American Society of Hematology (ASH) national meeting in December, found that patient with hemophilia who require routine prophylaxis may be more adherent to emicizumab than to previous factor or bypass agents. The study aimed to asses adherence rates and find potential​ predictors of nonadherence associated with emicizumab treatment. See full article at

Sangamo Shares Data for Gene Therapy Study on SB-525 for Hemophilia A

Sangamo Therapeutics and Pfizer announced updated follow-up results from their Phase 1/2 Alta study evaluating investigational SB-525 gene therapy in patients with severe hemophilia A. The data showed that it was generally well tolerated, and demonstrated sustained increased Factor VIII levels following treatment through to 44 weeks. See full article at

Updated MASAC Guidelines on Emergency Department Management Approved

The National Hemophilia Foundation's Medical and Scientific Advisory Council (MASAC) has issued an important new document on emergency department management, which was adopted by the NHF's Board of Directors on Dec 5, 2019. The document includes guidelines including triage, assessment, and diagnostics for patients who need to visit the emergency department. See full article at

Application deadline for NHF's National Youth Leadership Institute is Oct 31

The National Youth Leadership Institute (NYLI) is a program for young adults (18-24) in the bleeding disorder community to gain leadership experience, work with advocacy groups and non-profits, and deliver education to local NHF chapters. The application deadline is Oct 31, 2019. See full article at

FDA Approves Wilate for Hemophilia A in Adult and Adolescent Patients

The FDA has approved Octapharma's Wilate for the treatment of adults and adolescents with hemophilia A for routine prophylaxis to reduce the frequency of bleeding episodes, on demand treatment, and control bleeding episodes. See full article at

Genentech Issues Statement on Hemlibra Particles; MASAC Responds

Genentech issued a statement about the quality assurance of their manufacturing when they found unexpected particles in batches of Hemlibra. These particles were discovered to consist of protein and a non-toxic polymer. Both Genentech and MASAC issued statements explaining their findings and no adverse events are linked to the particulate matter found. See full article at

Freeline's Gene Therapy for Hemophilia B Shows Promising Phase 1/2 Results

Early data from Freeline's single infusion gene therapy for patients with hemophilia B, FLT180a, showed a rise in clotting factor IX to 40% with levels remaining stable for over a year. Freeline's experimental therapy uses a AAV capsid to deliver a functional version of human clotting factor. See full article at

Study Shows Hemophilia 3 Times as Prevalent in Men than Originally Thought

Researchers at McMaster University in Canada believe that three times more men in the world have hemophilia than previously believed. The study found that close to 1,125,000 men around the world have hemophilia and that 418,000 have the severe form of the disorder. See full article at

BioMarin Explains Phase 3 Results for Hemophilia A Gene Therapy Trial

BioMarin Pharmaceutical explained results from their Phase 3 valrox gene therapy trial. The results showed timing of steroid administration may have played a role in the initial interim results. A delay in starting steroids stems from the study's design, which calls for an on-demand use rather than prophylactic regimen​ that was used in Phase 2. See full article at

Hemlibra Given with Immune Tolerance Induction Safely Treats Children

A new study published in the journal Haemophilia shows that combining immune tolerance induction can help prevent development of anti-factor VIII inhibitors when used with Hemlibra treating children with severe hemophilia A.  See full article at