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October 14, 2019October 14, 2019

Novo Nordisk Partners with Bluebird Bio to Develop New Gene Therapies for Hemophilia, Other Disorders

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Novo Nordisk has entered into a collaboration agreement with Bluebird Bio to develop new therapies based on gene editing for a series of genetic disorders, including hemophilia. The companies ... Read more

October 11, 2019October 11, 2019

FDA Approves Wilate for Adults and Adolescents With Hemophilia A

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The U.S. Food and Drug Administration (FDA) has approved Wilate as a prophylactic (preventive) and on-demand treatment for bleeding episodes in adults and adolescents with hemophilia A. Wilate, developed ... Read more

October 9, 2019October 9, 2019

Muscle Rehabilitation Exercises Safe, Effective for People with Severe Hemophilia, Study Suggests

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Moderate muscle rehabilitation exercises — based on the use of elastic bands or training machines — are safe and effective for people with severe hemophilia receiving prophylactic (preventive) treatment, ... Read more

October 7, 2019October 7, 2019

Work, Relationship Issues Affect Health-related Quality of Life, Pain in Adults with Hemophilia B, Study Says

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Problems at work, limited engagement in recreational activities, and less relationship satisfaction contribute to a lower health-related quality of life (HQRoL) and greater pain severity, as reported by adults ... Read more

October 4, 2019October 4, 2019

DalcA, Under-skin Injectable Factor IX Prophylactic, Showing Potential in Phase 2 Trial, Catalyst Says

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A preventive treatment for hemophilia B called dalcinonacog alfa (DalcA) led to an increase of more than 12% in the levels of factor IX in two patients with severe disease treated in ... Read more

October 2, 2019October 2, 2019

New Gene Therapy Approach Led to Sustained Benefits in Mouse Model of Hemophilia A, Study Reports

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A gene therapy approach using a so-called antibody-drug conjugate (ADC) conditioning regimen led to safe and sustained production of factor VIII (FVIII) in platelets, and prevented joint bleeding in ... Read more

September 30, 2019September 30, 2019

Gene Therapy AMT-061 Provides Greater Factor IX Activity to Possibly Prevent Joint Disease, Study Reports

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The gene therapies AMT-060 and AMT-061 both restored coagulation factor IX levels in a primate model of hemophilia B, but AMT-061 resulted in greater coagulation activity at the same ... Read more

September 27, 2019September 27, 2019

NORD 2019 Rare Disease Summit Set for Oct. 21-22 in Washington, DC

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Next month’s annual conference of the National Organization for Rare Disorders (NORD) in Washington, D.C., couldn’t come at a better time, says Marshall Summar, MD, chairman of NORD’s board ... Read more

September 27, 2019September 27, 2019

Chinese Patients with Rare Diseases Lack Affordable, Accessible Treatment

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Health insurance is largely effective for Chinese patients with rare diseases such as hemophilia when it comes to being able to utilize healthcare services and buy medication. However, affordable ... Read more

September 25, 2019September 25, 2019

Hemlibra Use in Severe Hemophilia A Could Save $3 Billion Over 20 Years, Study Estimates

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Using Hemlibra (emicizumab) as prophylaxis lowers treatment costs considerably for people with hemophilia A compared to preventative factor (FVIII) treatment, according to an economic model study. The model predicts $47,814 in ... Read more

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