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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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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Clinical Trial Updates for Achondroplasia and Hemophilia A!
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Clinical Trial Updates for Achondroplasia and Hemophilia A!

BioMarin has just released an update on their achondroplasia and hemophilia clinical trials. Exciting things are in the works! Achondroplasia Update Achondroplasia causes short stature. A phase 3 trial working to…

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Preliminary Data from a Phase 1/2 Study of a Gene Therapy for Haemophilia A has been Shared
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Preliminary Data from a Phase 1/2 Study of a Gene Therapy for Haemophilia A has been Shared

Sangamo Therapeutics has shared details of their preliminary data from a clinical trial of the investigational drug SB-525 in patients with Haemophilia A. For more detailed information, you can read…

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Hemophilia Doesn’t Just Affect Men, Let’s Stop Talking like It Does
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Hemophilia Doesn’t Just Affect Men, Let’s Stop Talking like It Does

Hemophilia can affect anyone. Let’s make a few things clear: Women are carriers of hemophilia and pass the gene, but men determine the child’s gender. It’s not all on you,…

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Tips for a New Hemophilia Patient and New Caregiver: Reporting from the WFH Congress
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Tips for a New Hemophilia Patient and New Caregiver: Reporting from the WFH Congress

To the patient: Accepting your diagnosis doesn’t mean you can’t be mad about it. It simply means you understand that the reality of your life has changed and you know…

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