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presented at National Tay-Sachs Symposium Breakthroughs presented at National Tay-Sachs Symposium Sena-Esteves reports human gene therapy trials may begin next year Mark Shelton UMass Medical School Communications Sena-Esteves, Tay-Sachs      Miguel Sena-Esteves, PhD Breakthroughs in crossing the blood–brain barrier that could lead to new treatments for Tay-Sachs, Canavan and other neurodegenerative diseases were among the research findings presented at the National Tay-Sachs & Allied Diseases (NTSAD)

Radio Boston: Tay-Sachs expert talks about gene therapy trial Radio Boston: Tay-Sachs expert talks about gene therapy trial Jim Fessenden UMass Medical School Communications Radio Boston host Jessica Alpert interviewed Miguel Sena-Esteves, PhD, associate professor of neurology, and Carey Goldberg, WBUR Commonhealth reporter, for a segment on a human gene therapy trial for Tay-Sachs disease. Radio Boston, Tay-Sachs, Miguel Sena-Esteves   Miguel Sena-Esteves, PhD Radio Boston host Jessica Alpert interviewed

therapies based on UMass Medical School research discoveries, has announced that the FDA has lifted its clinical hold and cleared Axovant’s Investigational New Drug application to initiate a registrational study of gene therapy to treat patients with Tay-Sachs disease and Sandhoff disease.

Tay-Sachs experts interviewed on progress of gene therapy trials Tay-Sachs experts interviewed on progress of gene therapy trials Miguel Sena-Esteves talks about the promise of clinical trials Jim Fessenden UMass Medical School Communications Miguel Sena-Esteves, PhD, associate professor of neurology and a leading force behind the creation of the Tay-Sachs Gene Therapy Consortium, an international collaboration of scientists committed to translating current gene therapy results into a human clinical

Top story: Catherine’s story: Tay-Sachs disease Top story: Catherine’s story: Tay-Sachs disease UMass Chan Medical School Communications UMass Chan Medical School Communications Top story: UMass Chan Medical School has launched Rare Diseases, Real Stories, a six-episode podcast series. This is Catherine's story. It is available on all major streaming platforms. Top story: UMass Chan Medical School has launched Rare Diseases, Real Stories, a six-episode podcast series. This is Catherine's story. It

Foundation, a Toronto-based foundation dedicated to the development of gene therapy treatments for rare disease, has donated $1.4 million to UMass Medical School for the advancement of a Phase I/II clinical trial for the genetic disorder known as Tay-Sachs.

Thomas Feldborg and Daria Rokina, of Denmark, chose to enroll their baby daughter, Alissa, in an investigational gene therapy for the treatment of GM2 gangliosidosis, which causes Tay-Sachs and Sandhoff diseases, to “let hope conquer the fears,” according to a report in USA Today chronicling their journey.

In a piece written for The Conversation, Miguel Sena-Esteves, PhD, talks about research leading to the first ever gene therapy for Tay-Sachs disease and the launch of the Translational Institute for Molecular Therapeutics at UMass Chan.

Miguel Sena-Esteves tells the Washington Post how a rare breed of ancient sheep has played a key role in Tay-Sachs research.

Axovant Sciences, a Swiss company developing gene therapies for neurological diseases, has licensed exclusive worldwide rights for the development and commercialization of two novel gene therapy programs for Tay-Sachs and Sandhoff diseases from UMass Medical School.