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Spotlighting Rare Diseases Research Blog

UMass Chan Researcher Develops A New Gene Therapy for Children With Sandhoff Disease

Posted On: abr 28, 2021

A new gene therapy approach for Sandhoff disease developed by UMass Chan researcher Miguel Sena-Esteves is featured in a story in USA Today.

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Pilot Grant Program for Rare Disease Research Awardees

Posted On: mar 19, 2021

Two research scientists from UMass Chan Medical School — Kevin Donahue, MD and Marian Walhout, PhD — have been awarded funds for research on much-needed therapies for certain rare diseases...

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Early Discoveries on the Genetics and Treatment of Canavan Disease

Posted On: ago 1, 2019

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I began working in the area of rare disease research during the early years of my research training as a PhD student. This work was focused on molecular genetics, disease gene cloning, and genetic diagnosis of several rare disorders of the central nervous system (CNS), including Canavan disease, Hurler syndrome, and Hunter syndrome...

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