×
About 1,363 results

ALLMedicine™ Hemophilia B Center

Research & Reviews  390 results

A Phase 1/2 Study of SHP648, an Adeno-Associated Viral Vector for Gene Transfer in Hemophilia B Subjects
https://clinicaltrials.gov/ct2/show/NCT04394286

May 19th, 2022 - 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...

Open-Label Extension Study of Marstacimab in Hemophilia Participants With or Without Inhibitors
https://clinicaltrials.gov/ct2/show/NCT05145127

May 17th, 2022 - Study B7841007 is an open-label extension study to assess the long-term safety, tolerability, and efficacy of prophylaxis treatment with marstacimab in participants who did not require "Early Termination" from the Phase 3 Study B7841005. Approxima...

Management and outcomes of mild hemophiliacs and hemophilia carriers during pregnancy a...
https://doi.org/10.1080/14767058.2022.2051699
The Journal of Maternal-fetal & Neonatal Medicine : the O... Lim HI, DeSancho MT

Apr 14th, 2022 - Pregnancy, peripartum management, and outcomes of mild hemophiliacs and hemophilia carriers in the United States are not well established. To describe the management and outcomes of mild hemophiliacs and hemophilia carriers during assisted concept...

Structural, functional, and immunogenicity implications of F9 gene recoding.
https://doi.org/10.1182/bloodadvances.2022007094
Blood Advances; Katneni U, Alexaki A et. al.

Apr 13th, 2022 - Hemophilia B is a blood clotting disorder caused by deficient activity of coagulation factor IX (FIX). Multiple recombinant FIX proteins are currently approved to treat hemophilia B and several gene therapy products are currently being developed. ...

Trial of AAV5-hFIX in Severe or Moderately Severe Hemophilia B
https://clinicaltrials.gov/ct2/show/NCT02396342

Apr 8th, 2022 - This study evaluates how safe gene therapy treatment with AAV5-hFIX is in adult patients with severe or moderately severe hemophilia B and severe bleeding type.

see more →

Drugs  24 results see all →

Clinicaltrials.gov  27 results

A Phase 1/2 Study of SHP648, an Adeno-Associated Viral Vector for Gene Transfer in Hemophilia B Subjects
https://clinicaltrials.gov/ct2/show/NCT04394286

May 19th, 2022 - 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...

Open-Label Extension Study of Marstacimab in Hemophilia Participants With or Without Inhibitors
https://clinicaltrials.gov/ct2/show/NCT05145127

May 17th, 2022 - Study B7841007 is an open-label extension study to assess the long-term safety, tolerability, and efficacy of prophylaxis treatment with marstacimab in participants who did not require "Early Termination" from the Phase 3 Study B7841005. Approxima...

Trial of AAV5-hFIX in Severe or Moderately Severe Hemophilia B
https://clinicaltrials.gov/ct2/show/NCT02396342

Apr 8th, 2022 - This study evaluates how safe gene therapy treatment with AAV5-hFIX is in adult patients with severe or moderately severe hemophilia B and severe bleeding type.

Long-Term Safety, Tolerability, and Efficacy of DTX101 (AAVrh10FIX) in Adults With Moderate/Severe to Severe Hemophilia B
https://clinicaltrials.gov/ct2/show/NCT02971969

Jan 6th, 2022 - Hemophilia B is an X-linked recessive genetic bleeding disorder caused by mutations in the factor IX (FIX) gene. FIX is produced in the liver and is critical for fibrin clot formation. Hemophilia B is characterized by frequent, spontaneous interna...

Safety and Tolerability of VGB-R04 in Patients With Haemophilia B
https://clinicaltrials.gov/ct2/show/NCT05152732

Dec 10th, 2021 - Hemophilia B is a genetic bleeding disorder caused by pathogenic variants (eg, mutations, deletion) in the FIX gene. HB patients have frequent and potentially life-threatening bleeding and often develop progressive physical disability and pain fro...

see more →

News  103 results

Gene Therapy: A 'One and Done' Hemophilia B Treatment?
https://www.medscape.com/viewarticle/968936

Feb 22nd, 2022 - Nearly all patients treated with a one-time gene therapy shot for hemophilia B (HB) were able to discontinue full-dose bleeding prophylaxis, a new industry-funded phase 3 study finds, and annual bleed rates (ABR) fell by 64% after the lead-in peri...

Gene therapy: A ‘one and done’ hemophilia B treatment?
https://www.mdedge.com/hematology-oncology/article/251916/bleeding-disorders/gene-therapy-one-and-done-hemophilia-b
Randy Dotinga

Feb 17th, 2022 - Nearly all patients treated with a one-time gene therapy shot for hemophilia B (HB) were able to discontinue full-dose bleeding prophylaxis, a new industry-funded phase 3 study finds, and annual bleed rates (ABR) fell by 64% after the lead-in peri.

Health Costs Over 25 Times Higher for Patients With Hemophilia B
https://www.medscape.com/viewarticle/951014

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...

Bleeding Disorder Diagnoses Delayed by Years in Girls and Women
https://www.medscape.com/viewarticle/947192

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...

Safety profiles of hemophilia agents vary widely
https://www.mdedge.com/hematology-oncology/article/236344/bleeding-disorders/safety-profiles-hemophilia-agents-vary-widely
Neil Osterweil

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. “.

see more →

Patient Education  1 results see all →