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January 24, 2020January 24, 2020

Poor Joint Health Linked to Lower Self-Esteem in Adolescents with Hemophilia, Study Suggests

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Poor joint health is associated with a higher risk of low self-esteem in adolescents with hemophilia, a study has found. The study, “Assessment of Self-Image With the Offer Self-Image ... Read more

January 22, 2020January 22, 2020

Hemlibra Used to Prevent Bleeds Can Aid Joint Health in Hemophilia A, Trial Data Show

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Prophylactic, or preventive, use of Hemlibra (emicizumab) promotes joint health in people with hemophilia A, according to a new analysis of data from a Phase 3 trial. These findings were ... Read more

January 20, 2020January 20, 2020

Valrox, Potential Hemophilia A Gene Therapy, May Cost Up to $3 Million, BioMarin Says

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BioMarin is considering pricing its hemophilia A gene therapy Valrox (valoctocogene roxaparvovec), should it be approved, at $2 million to $3 million. That range would make Valrox the world’s ... Read more

January 17, 2020January 17, 2020

Risk of Inhibitors with Nuwiq Similar to Plasma-derived Products in Untreated Hemophilia A Patients, Trial Shows

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Adults and children with severe hemophilia A who are receiving Nuwiq (recombinant human coagulation factor VIII) as their first replacement therapy have a similar risk of developing inhibitors as ... Read more

January 15, 2020January 15, 2020

ASC Therapeutics Partners with Vigene for Manufacturing Support for Hemophilia A Gene Therapy Program

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ASC Therapeutics is partnering with Vigene Biosciences to support the manufacturing process of its current and future gene therapy clinical platforms — including its ongoing hemophilia A program. “We ... Read more

January 13, 2020January 13, 2020

Generation Bio Raises $110 Million for Non-viral Gene Therapy for Hemophilia A, Other Diseases

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Generation Bio said it received $110 million in funding to bring its innovative, non-viral gene therapy for lead programs hemophilia A and phenylketonuria into investigational new drug-enabling clinical trials. Gene ... Read more

January 10, 2020January 10, 2020

AMT-061 Increases FIX Activity, Prevents Bleeds in Severe Hemophilia B Patients, Phase 2b Data Show

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A single infusion of AMT-061 (etranacogene dezaparvovec), uniQure’s experimental gene therapy for the treatment of hemophilia B, led to increased and sustained activity of clotting factor IX (FIX), according ... Read more

January 8, 2020January 8, 2020

BioMarin’s Gene Therapy, Found to Reduce Bleeding Events in Hemophilia A, Submitted for U.S. Approval

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BioMarin Pharmaceuticals’ investigational gene therapy valoctocogene roxaparvovec safely and significantly reduced bleeding events — including in target joints — and the use of prophylactic clotting factor VIII in adults with ... Read more

January 6, 2020January 6, 2020

Pfizer Taking over Development of Potential Gene Therapy SB-525, Advancing into Phase 3 Trials

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Sangamo Therapeutics has handed over the development of SB-525, a gene therapy for hemophilia A, to Pfizer, which will now advance the therapy into Phase 3 clinical trials. Pfizer ... Read more

January 3, 2020January 3, 2020

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Poor Joint Health Linked to Lower Self-Esteem in Adolescents with Hemophilia, Study Suggests

Hemlibra Used to Prevent Bleeds Can Aid Joint Health in Hemophilia A, Trial Data Show

Valrox, Potential Hemophilia A Gene Therapy, May Cost Up to $3 Million, BioMarin Says

Risk of Inhibitors with Nuwiq Similar to Plasma-derived Products in Untreated Hemophilia A Patients, Trial Shows

ASC Therapeutics Partners with Vigene for Manufacturing Support for Hemophilia A Gene Therapy Program

Generation Bio Raises $110 Million for Non-viral Gene Therapy for Hemophilia A, Other Diseases

AMT-061 Increases FIX Activity, Prevents Bleeds in Severe Hemophilia B Patients, Phase 2b Data Show

BioMarin’s Gene Therapy, Found to Reduce Bleeding Events in Hemophilia A, Submitted for U.S. Approval

Pfizer Taking over Development of Potential Gene Therapy SB-525, Advancing into Phase 3 Trials

Top 10 Hemophilia Stories of 2019

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