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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Press Release: uniQure Announces First Patient Treated in HOPE-B Pivotal Trial of AMT-061 in Patients with Hemophilia B

Full Patient Enrollment in Study Expected by Year-End 2019 LEXINGTON, Mass. and AMSTERDAM, The Netherlands, Feb. 04, 2019 -- uniQure N.V. (NASDAQ: QURE), a leading gene therapy company advancing transformative therapies for…

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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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Why We Need to Focus on Adherence, Not Compliance: Reporting from the WFH Congress
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Why We Need to Focus on Adherence, Not Compliance: Reporting from the WFH Congress

We often hear the term noncompliance when talking about patient-doctor relationships for individuals with hemophilia. In essence, it means the hemophilia patient hasn’t done what the doctor has asked them…

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UniQure Publishes Phase I/II Trial Results of AMT-060 Gene Therapy in Treating Hemophilia B
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UniQure Publishes Phase I/II Trial Results of AMT-060 Gene Therapy in Treating Hemophilia B

Pharmaceutical company and gene therapy leader, uniQure, published promising data from their current phase I/II trial testing out a gene therapy called AMT-060 in patients with serious cases of hemophilia B.…

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