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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Enrollment Finishes Early for a Late Stage Hemophilia B Gene Therapy Clinical Trial
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Enrollment Finishes Early for a Late Stage Hemophilia B Gene Therapy Clinical Trial

According to a story from Hemophilia News Today, the drug developer uniQure has completed its goals for patient enrollment ahead of time for is phase 3 clinical trial. This clinical…

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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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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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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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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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Sanofi Acquires Hemophilia Treatment Developer Bioverativ for $11.6 Billion
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Sanofi Acquires Hemophilia Treatment Developer Bioverativ for $11.6 Billion

A huge deal is underway as Sanofi looks to acquire Bioverativ for $11.6 billion with hope to enhance and strength their rare disease presence, reports Genetic Engineering & Biotechnology News.…

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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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National Hemophilia Foundation’s 69th Annual Meeting Wrap-Up!
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National Hemophilia Foundation’s 69th Annual Meeting Wrap-Up!

Patient Worthy attended the National Hemophilia Foundation's 69th Annual meeting in Chicago and we are thrilled to report on some of the exciting new developments for patients with bleeding disorders.…

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