Couple Adopts Child with Hemophilia
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Couple Adopts Child with Hemophilia

Monica and Josh Poynter have recently adopted a nine-year-old boy, Trey, from China. They felt a connection to the boy as he has hemophilia type A, the same rare bleeding…

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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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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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