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December 20, 2019December 20, 2019

Takeda Presents Real-World Data on Cost-Effectiveness and Long-term Benefits of Adynovate and Feiba

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Takeda has presented new real-world data to support the cost-effectiveness and persistent benefits to patients of its hemophilia A and B therapies: Adynovate and Feiba ... Read more

December 18, 2019December 18, 2019

Takeda Presents Early Data About Improving Efficiency of Hemophilia A and B Gene Therapy

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Takeda has presented early data on the prevalence of — and a possible solution for — one of gene therapy’s main hurdles: the development of an immune reaction against the ... Read more

December 16, 2019December 16, 2019

Takeda, Virgin Pulse Team to ‘Challenge’ Hemophilia Patients into Adopting Healthy Lifestyles

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To help promote healthy lifestyle changes, the pharmaceutical company Takeda has enrolled some 900 patients with bleeding disorders like hemophilia in the Virgin Pulse Global Challenge. The annual well-being Global ... Read more

December 11, 2019December 11, 2019

Gene Therapy Takes Center Stage at 2019 NORD Summit

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With so much recent publicity surrounding gene therapy, it’s no surprise that the topic was a major focus of the recent 2019 NORD Rare Diseases & Orphan Products Breakthrough ... Read more

December 11, 2019December 11, 2019

Bayer, Children’s Hospital of Philadelphia Working on Non-replacement Hemophilia Therapies

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Bayer has entered into a three-year research partnership with the Children’s Hospital of Philadelphia to develop small molecule non-replacement therapies as a first-line and less burdensome oral treatment for ... Read more

December 10, 2019December 13, 2019

New Early Data Support Catalyst’s MarzAA in Treating Acute Bleeds in Hemophilia

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New preclinical data on one of Catalyst Biosciences‘ lead candidate therapies — marzeptacog alfa activated (MarzAA), an engineered clotting Factor VIIa (FVIIa) given as an under-the-skin injection — support its potential ... Read more

December 9, 2019December 18, 2019

Enzyre and Takeda to Develop Coagulation Test that Patients Can Use at Home

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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. Under the collaboration agreement, ... Read more

December 6, 2019December 6, 2019

HHS Secretary Alex Azar Touts White House Efforts to Cure Rare Diseases

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Despite skyrocketing healthcare costs, President Trump is committed to protecting the 30 million or so Americans with rare diseases and ensuring timely, affordable access to lifesaving treatments, the nation’s ... Read more

December 6, 2019December 6, 2019

First 2 Adults with Severe Hemophilia A Respond Well to Gene Therapy in Clinical Trial

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Bayer’s investigational gene therapy BAY 2599023 safely and effectively increased the levels of clotting factor VIII (FVIII) and prevented or lessened bleeding in the first two people with severe hemophilia A treated ... Read more

December 4, 2019December 4, 2019

Insurer Highmark Forges New Hemophilia Partnership Focused on Treatment, Management

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As part of a new partnership focused on hemophilia treatment and management, members of insurer Highmark will receive medication exclusively from Option Care Health, Soleo Health, and the Hemophilia ... Read more

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