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Gene Therapy Trial for GSD1a Progress.

Trials for a gene therapy treatment for GSD1a are continuing and have now reached phase III


Following David Weinstein’s departure from UConn, Dr Rebecca Riba-Wolman has taken the lead in and progressed the work. The ultimate hope is that a treatment will be available for all those that have GSD1a, following the process of review and approval.

Please follow the link for more insight on this development.

Link to UConn Today