Note: This story was updated on June 24, 2020, to clarify that higher daily doses, rather than long-term exposure, were thought to contribute to the loss of benefit. The headline was also changed to more accurately reflect the mechanism by which therapy effectiveness may be affected. Higher daily doses of…
PTC Therapeutics is delaying by at least three months the submission of an investigational new drug application requesting that the gene therapy candidate GT-AS enter clinical trials for Angelman syndrome. The delay is a consequence of the COVID-19 pandemic, which has affected many preclinical studies and…
The U.S. Food and Drug Administration (FDA) has given fast track designation to GeneTx Biotherapeutics and Ultragenyx‘s investigational therapy GTX-102 for Angelman syndrome. Fast track is given to speed the development and review of a treatment, facilitating discussions with the…
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