Penn and CHOP researchers want to develop urea cycle disorder treatments using CRISPR gene-editing therapy. Get unlimited access to Inquirer.com and The Inquirer App, plus 5 articles each month to ...
Promising results from a small clinical trial highlight a growing interest in designing gene-editing treatments for common ...
A sponsor will have to demonstrate success with "with several consecutive patients with different bespoke therapies" to ...
In a highly anticipated article, FDA officials Vinay Prasad, M.D., and Martin Makary, M.D., outlined a novel regulatory ...
Despite the best efforts of modern medicine, Huntington’s disease is a condition that still comes with a tragic prognosis.
Scientists at St. Jude Children's Research Hospital and Northwestern University identified a previously unknown treatment ...
With a snip of a gene, doctors may one day permanently lower dangerously high cholesterol, possibly removing the need for ...
Sanofi SA (NASDAQ:SNY) on Wednesday revealed topline data from the ElevAATe phase 2 study of efdoralprin alfa (SAR447537, formerly known as INBRX-101), for alpha-1 antitrypsin deficiency (AATD) ...
NEW YORK – A gene therapy for heart failure being advanced by Bayer's AskBio subsidiary has shown a promising safety profile and early signs of efficacy in a small first-in-human Phase I trial, ...
Optogenetics shows promise in improving mobility and object recognition for patients with retinitis pigmentosa and Stargardt disease. Stephen Tsang, MD, PhD, professor at Columbia University and ...
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