Doctors harvest stem cells from the patient’s bone marrow and then send them off to a lab, where CRISPR is used to turn up the production of another, functional version of haemoglobin. This version is ...
After asset purchase from Travere, Mirum's Ctexli crosses FDA finish line in rare metabolic disorder
The approval makes chenodiol—already cleared for decades to treat radiolucent gallstones under the brand name Chenodal—the ...
Combining an existing small-molecule protein therapy called tumor necrosis factor related apoptosis-inducing ligand (TRAIL) ...
With PEPFAR seemingly now focused primarily on the treatment of existing patients, at the expense of prevention, clinicians like Nomathemba Chandiwana, a physician-scientist at the Desmond Tutu Health ...
Of the first four experimental ALS treatments tested in the HEALEY platform trial, CNM-Au8 and pridopridine showed some promising results.
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