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In section: UMass Chan News
In subsection: Articles
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)
Last modified: Dec 27, 2013
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In section: UMass Chan News
In subsection: Articles
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
Last modified: Dec 16, 2013
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In section: UMass Chan News
In subsection: Articles
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.
Last modified: Nov 09, 2020
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In section: UMass Chan News
In subsection: Articles
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
Last modified: Dec 16, 2013
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In section: UMass Chan News
In subsection: Articles
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
Last modified: Dec 15, 2025
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In section: UMass Chan News
In subsection: Articles
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.
Last modified: Nov 06, 2018
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In section: UMass Chan News
In subsection: Articles
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.
Last modified: Apr 27, 2021
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In section: UMass Chan News
In subsection: Articles
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.
Last modified: Feb 14, 2022
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In section: UMass Chan News
In subsection: Articles
Miguel Sena-Esteves tells the Washington Post how a rare breed of ancient sheep has played a key role in Tay-Sachs research.
Last modified: May 24, 2016
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In section: UMass Chan News
In subsection: Articles
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.
Last modified: Dec 13, 2018