FDA launches Rare Disease Evidence Principles to guide therapy reviews, offering new pathways for approval while raising ...

And it has paved the way for other therapeutic strategies, including the first gene therapy delivered through skin grafts, ...

In the last few years, Gene and Cell Therapy research has emerged as one of the most promising technologies for treating ...

Gene therapy represents a paradigm shift in modern medicine, offering unprecedented opportunities to combat diseases at their genetic origin.

An expert discusses how gene therapy represents a breakthrough treatment approach for BCG-unresponsive patients, offering quarterly dosing that reduces treatment burden while showing promising ...

Although there is a wide range in how Down syndrome may present in different patients, it can sometimes be a devastating disease. There are no treatments for the condition. This work has shown that it ...

A recent story in the New York Times about gene therapy for sickle-cell trait lays bare the tough decision for benefits administrators — paying for this treatment is extraordinarily expensive, the ...

For years, Stephanie McCabe and Andre Doucette accepted that their six-year-old son Emmett would die young. Now a possible treatment for Sanfilippo syndrome is changing everything for the Saskatoon ...

Single-Assay Evaluation of AAV Capsid Quality: Novel Digital PCR Approaches Advance Gene Therapy Research and Production Bio-Rad’s ddPCR-enhancing technology provides genome titer, capsid titer ...

Cancer immunotherapy has advanced the field of medicine and cancer care. Specifically, immunotherapy refers to the targeted ...

The type of estradiol-based hormone therapy taken during and after menopause, such as patches or pills, may be associated ...