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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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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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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A Letter to Parents on Transitioning from Pediatric to Adult Care in Hemophilia: Reporting from the WFH Congress
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A Letter to Parents on Transitioning from Pediatric to Adult Care in Hemophilia: Reporting from the WFH Congress

Dear Parents, You need to understand that you are taking on a completely new role in your child’s life. You will no longer be controlling their hemophilia for them. It’s…

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