ALLMedicine™ Hemophilia B Center

Nov 11th, 2021 - Gene therapy is a promising treatment for protein deficiency disorders such as hemophilia B. However, low tissue selectivity and efficacy are limitations of systemic vector delivery. The authors hypothesized that selective transfection of rat supe...

Nov 9th, 2021 - This is an open-label, single-dose, multi-center, multinational trial to demonstrate the efficacy of AMT-061 and to further describe its safety profile. The study drug is identified as AAV5-hFIXco-Padua (AMT- 061). AMT-061 is a recombinant adeno-a...

Oct 26th, 2021 - This prospective cohort study will use qualitative methods, specifically semi structured interviews to evaluate knowledge, beliefs, and attitude about gene therapy in patients 12 years and older with a diagnosis of moderate and severe hemophilia B...

Oct 24th, 2021 - Pluripotent stem cell-derived hepatocytes differentiated in monolayer culture are known to have more foetal than adult hepatocyte characteristics. If numerous studies tend to show that this immature phenotype might not necessarily be an obstacle t...

Oct 16th, 2021 - Hemophilia B is a bleeding disorder, caused by a factor IX (FIX) deficiency. Recently, FIX concentrates with extended half-life (EHL) have become available. Prophylactic dosing of EHL-FIX concentrates can be optimized by assessment of individual p...

Nov 9th, 2021 - This is an open-label, single-dose, multi-center, multinational trial to demonstrate the efficacy of AMT-061 and to further describe its safety profile. The study drug is identified as AAV5-hFIXco-Padua (AMT- 061). AMT-061 is a recombinant adeno-a...

Oct 26th, 2021 - This prospective cohort study will use qualitative methods, specifically semi structured interviews to evaluate knowledge, beliefs, and attitude about gene therapy in patients 12 years and older with a diagnosis of moderate and severe hemophilia B...

Sep 23rd, 2021 - Study APVO101-903 is a Phase 3/4, single arm, open-label clinical trial. The purpose of the study is to evaluate pharmacokinetics (PK), safety, and efficacy of APVO101 prophylaxis in severe or moderately severe hemophilia B subjects < 12 years of ...

Aug 13th, 2021 - This study will evaluate the efficacy and safety of PF-06838435 (a gene therapy drug) in adult male participants with moderately severe to severe hemophilia B (participants that have a Factor IX circulating activity of 2% or less). The gene therap...

Jul 1st, 2021 - This study will consists of 3 dose cohorts with 2-7 participants in each of the three ascending dose cohorts. Initially 2 participants will be dosed in Cohort 1, followed by dosing of up to 5 additional participants if the cohort is expanded. Part...

Aug 4th, 2021 - Recombinant factor IX Fc fusion protein was effective, both as prophylaxis and in the treatment of bleeding episodes in previously untreated boys (< 18 years of age) with hemophilia B, according to Beatrice Nolan, MD, of Children’s Health Ireland.

May 12th, 2021 - As the burden of hemophilia B in patients increases from mild to severe forms of the disease, the already high economic cost of treatment rises significantly, according to a large retrospective database study. Researchers developed four profile ca...

Mar 10th, 2021 - Diagnosis of bleeding disorders in girls and women can lag behind diagnosis in boys and men by more than a decade, meaning needless delays in treatment and poor quality of life for many with hemophilia or related conditions. "There is increasing a...

Feb 24th, 2021 - Despite their similar functions, each current and emerging therapy for treating hemophilia has a unique safety profile, and each needs to be weighed apart from agents both within and outside its pharmacologic class, a hemophilia specialist said. “.

Dec 18th, 2020 - NEW YORK (Reuters Health) - An investigational gene therapy for hemophilia B substantially increased production of the blood-clotting protein factor IX and reduced the number of bleeding episodes and need for prophylactic therapy in a phase-3 tria...