News

March 10, 2021March 10, 2021

Roctavian, Potential Hemophilia A Gene Therapy, Get Fresh FDA Support

News

The U.S. Food and Drug Administration (FDA) has given regenerative medicine advanced therapy (RMAT) designation to Roctavian, an investigational gene therapy by BioMarin Pharmaceutical for severe hemophilia A. The designation is “a ... Read more

March 8, 2021March 7, 2021

Pink, Green, Blue and Purple Lights Illuminated World for Rare Disease Day

News, syndicated

Many diseases have their own awareness color — breast cancer is pink, muscular dystrophy is green, and AIDS is red, for example — but what’s the significance of pink, green, ... Read more

March 8, 2021March 8, 2021

Factor VII Treatment Eptacog Beta Now Under Review in Europe

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The European Medicines Agency (EMA) has agreed to review an application by Laboratoire Francais du Fractionnement et des Biotechnologies, a French biopharmaceutical known as LFB, requesting the approval of eptacog ... Read more

March 5, 2021March 5, 2021

Events Raise Visibility, Support of Bleeding Disorders During Awareness Month

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Activities are underway to mark Bleeding Disorders Awareness Month, set aside each March to call attention to such disorders as hemophilia and von Willebrand disease, and the more than three ... Read more

March 3, 2021March 3, 2021

Experts Identify Unique Hemophilia Challenges, Propose Ways to Overcome

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People with mild to moderate hemophilia, in particular women with the disease, face unique challenges, many that are specifically linked to a lack of information and insufficient engagement with other ... Read more

March 1, 2021March 1, 2021

Fitusiran Dosing in Trials Reduced to Lower Blood-clot Risk

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Sanofi has reduced the dosing of its investigational treatment fitusiran in clinical trials to lower the risk of blood clots in people with hemophilia, according to a joint statement representing ... Read more

February 26, 2021February 26, 2021

FDA Partially Rescinds Indications for BeneFIX and Ixinity

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Owing to conflicting market exclusivity rights, the U.S. Food and Drug Administration (FDA) has partially rescinded its decision to approve BeneFIX and Ixinity for additional indications both therapies received last year ... Read more

February 25, 2021February 25, 2021

Rare Disease Day ‘Patient Hero’ Raises Awareness Through Fashion

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Diagnosed with sickle cell disease as a 6-month-old, Tristan Lee has faced a lot of challenges over his 37 years of life. But from a young age, he also learned ... Read more

February 24, 2021February 24, 2021

Low Risk of Developing Inhibitors Found for Nuwiq Patients

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Treatment with Octapharma‘s Nuwiq is associated with a low risk of developing inhibitors — antibodies that prevent the medication from working properly — in previously untreated patients (PUPs) with hemophilia ... Read more

February 23, 2021February 23, 2021

Rare Disease Day at NIH, Set for March 1, Growing Year by Year

News, syndicated

Rare Disease Day at NIH, organized by the National Institutes of Health (NIH) and taking place on March 1, will feature panel discussions, patient stories, research updates, TED-style talks, and a ... Read more