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Gene therapy fails for Danish child treated for Sandhoff disease - Vimarsana News

Gene therapy fails for Danish child treated for Sandhoff disease

Alissa Feldborg, a 3-year-old with an ultra rare genetic disease called Sandhoff, died this month after participating in a gene therapy trial.

An experimental gene therapy was little Alissa's only hope. Now, instead of certain death, she faces an uncertain future. - Vimarsana News

An experimental gene therapy was little Alissa's only hope. Now, instead of certain death, she faces an uncertain future.

An experimental gene therapy was little Alissa's only hope. Now, instead of certain death, she faces an uncertain future. Karen Weintraub, USA TODAY © Feldborg family photo Alissa Feldborg BOSTON — Thomas Feldborg and Daria Rokina set off nearly every afternoon to explore this city. They leave from their hotel in the Charlestown neighborhood, pushing their baby carriage, some days heading deep into downtown, others choosing a path along the Charles River.  Every few minutes, Rokina stops to peek inside and check on 16-month-old Alissa. She gently rubs Alissa's chee...

Source: msn.com
Gene therapy offers hope for Danish girl, but uncertainty lies ahead - Vimarsana News

Gene therapy offers hope for Danish girl, but uncertainty lies ahead

12:12 pm UTC Apr. 25, 2021 BOSTON — Thomas Feldborg and Daria Rokina set off nearly every afternoon to explore this city. They leave from their hotel in the Charlestown neighborhood, pushing their baby carriage, some days heading deep into downtown, others choosing a path along the Charles River.  Every few minutes, Rokina stops to peek inside and check on 16-month-old Alissa. She gently rubs Alissa's cheek and coos a few soothing words, making sure the little girl is warm enough in her yellow snowsuit and adjusting her sparkly unicorn earmuffs.  Alissa rests better in the outsid...