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June 5, 2019June 5, 2019

Ratio Between FVIII and Von Willebrand Factor Predicts Relapse in Patients with Acquired Hemophilia A, Study Reveals

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The ratio between the blood clotting protein factor VIII (FVIII) and the von Willebrand factor (VWF) may be a reliable biomarker of recovery and relapse in patients with acquired ... Read more

June 3, 2019June 3, 2019

Esperoct Stabilizes/Improves Health-related Quality of Life of Patients With Severe Hemophilia A, Study Suggests

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Treatment with Esperoct led to stabilization and/or improvement of health-related quality of life (HRQoL) in patients with severe hemophilia A from all age groups, a study suggests. The study, ... Read more

May 31, 2019May 31, 2019

BioMarin’s Gene Therapy for Severe Hemophilia Yields Sustained Benefit, Data Show

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BioMarin Pharmaceutical’s investigative gene therapy, called valoctocogene roxaparvovec, is capable of sustained control of bleeding rate requiring factor VIII infusions by at least 92% in adults with severe hemophilia A, ... Read more

May 29, 2019June 3, 2019

Bayer Commits to 5-Year Pact with WFH to Help Hemophilia Patients in Underserved Countries

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A new five-year alliance between Bayer and the World Federation of Hemophilia (WFH) Humanitarian Aid Program will deliver training, education and treatments to healthcare professionals in more than 60 ... Read more

May 27, 2019May 27, 2019

Agency Unveils RaDaR to Help Patient Groups Develop Rare Disease Registries

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RaDaR, the catchy new name for the U.S. government-run Rare Diseases Registry Program, aims to help patient advocacy groups with limited resources build their own disease registries. The site ... Read more

May 24, 2019May 24, 2019

Web-Based Calculator Helps Physicians to Finetune Dosage of Hemlibra for Hemophilia A Patients

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BrightInsight has launched a web-based dosing calculator to help physicians select the correct loading and maintenance dose of the treatment Hemlibra according to the body measurements of their patients ... Read more

May 22, 2019May 22, 2019

Decision-making for Hemophilia Treatment Differs Between US and UK Doctors, Study Finds

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U.S. doctors caring for people with hemophilia are more likely to make treatment decisions based on patient preferences than doctors in the U.K., who tend to be more influenced ... Read more

May 17, 2019May 17, 2019

Candidate Cell Therapy Controls Bleeding in Mouse Model of Hemophilia A

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Sigilon Therapeutics’ candidate cell therapy for hemophilia A, called SIG-001, delivers sustained production of factor VIII for over six months and corrects bleeding in a hemophilia A mouse model. ... Read more

May 16, 2019May 20, 2019

ASC Therapeutics Licenses Tech from Expression Therapeutics to Advance Gene Therapies

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ASC Therapeutics has gotten exclusive rights to Expression Therapeutics‘ technology to develop liver-targeting hemophilia A gene therapies. “We have a common mission with Expression Therapeutics — to develop novel ... Read more

May 15, 2019

Rare Diseases Constitute a ‘Public Health Issue,’ NCATS Director Warns

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With 250 rare diseases newly identified every year, scientists can barely keep up — even as the healthcare system fails millions of Americans whose rare diseases have already been ... Read more

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