BioMarin Holds Clinical Study to Determine Antibodies that Might Impact Gene Therapy
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BioMarin Holds Clinical Study to Determine Antibodies that Might Impact Gene Therapy

Last month, biotechnology company BioMarin sought approval for an investigational gene therapy (valoctocogene roxaparvovec) for patients with hemophilia A. However, their approval was denied. Instead, the company was told that…

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Recall of Bleeding Disorder Drug Leaves Patients with Unanswered Questions
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Recall of Bleeding Disorder Drug Leaves Patients with Unanswered Questions

According to a story from hemophilia.org, the drug company Ferring Pharmaceuticals issued a recall of the nasal spray drug desmopressin (marketed as Stimate) on July 21st, 2020. This product is…

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Mouse Model of Gene Therapy is Shown to be Effective Against Hemophilia A
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Mouse Model of Gene Therapy is Shown to be Effective Against Hemophilia A

By Danielle Bradshaw from In The Cloud Copy The cells of hemophilia A patients were taken and genetically modified so that they would create an active clotting factor VIII (or…

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New Clinical Expectations Needed to Treat Hemophilia
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New Clinical Expectations Needed to Treat Hemophilia

The medical realm is changing - but how does that impact patient care and clinical expectations? The National Hemophilia Foundation discusses how clinical expectations for patients with hemophilia don't necessarily…

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Certain Proteins May Predict Joint Bleeding in Patients with Hemophilia
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Certain Proteins May Predict Joint Bleeding in Patients with Hemophilia

  Patients with hemophilia may experience sudden bleeding, joint pain and inflammation, and deep bruising. But according to Hemophilia News, two proteins may be the cause. Researchers found that patients…

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The Institute for Gene Therapies is Working to Improve Policy and Access for Charcot-Marie-Tooth Disease and Others
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The Institute for Gene Therapies is Working to Improve Policy and Access for Charcot-Marie-Tooth Disease and Others

The Institute for Gene Therapies (IGT) is a new development, aimed at improving the speed of development and accessibility of gene therapies. Gene Therapy Many rare diseases are caused by…

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Latest Data Release From Clinical Trial Suggests That Hemophilia A Gene Therapy Isn’t Far Off
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Latest Data Release From Clinical Trial Suggests That Hemophilia A Gene Therapy Isn’t Far Off

According to a story from biopharmadive.com, the race to a hemophilia A gene therapy continues to tighten following a recent release of early data from Sangamo Therapeutics and Pfizer. The…

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Severe Hemophilia A Patients May Soon See New  Gene Therapy Treatment Option
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Severe Hemophilia A Patients May Soon See New Gene Therapy Treatment Option

By Caitlin Seida from In The Cloud Copy People living with the severe hemophilia A may soon see a new treatment option available thanks to the work of BioMarin Pharmaceuticals.…

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The Drug Concizumab Appears Effective in Both Hemophilia A and B

According to a story from MD Edge, a phase 2 clinical trial testing the drug concizumab as a treatment for hemophilia A and hemophilia B demonstrated substantial capability in reducing…

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Experimental Hemophilia A Drug Loses Effectiveness Over Time, Efficacy Questionable After 8th Year

According to a publication from Reuters, based on early clinical data, American biotechnology company BioMarin Pharmaceuticals believes that a single injection of its experimental hemophilia A drug could relieve symptoms…

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Preventive Use of Extended Half-Life Therapies Provides Enhanced Benefit for Hemophilia A Patients
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Preventive Use of Extended Half-Life Therapies Provides Enhanced Benefit for Hemophilia A Patients

According to a story from BioSpace, a recent study revealed that hemophilia A patients were less likely to experience bleeds when they used new, extended half-life therapies on a preventive…

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Updates From Spark Highlight the Potential of Gene Therapy for Treating Hemophilia

According to a story from Hemophilia News Today, the drug developer Spark Therapeutics, Inc., has recently released several updates in regards to a number of its experimental gene therapies that…

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