Gene therapies have the potential to cure some diseases, but they are extraordinarily expensive. Location can also be a big hurdle for patients seeking this specialized care.
gene therapy
Posted inNPR
The FDA creates a quicker path for gene therapies
The Food and Drug Administration aims to evaluate treatments for rare diseases based on plausible evidence that they would work — without requiring a clinical trial first.
Posted inNPR
Sickle cell gene therapies roll out slowly
It’s been almost a year since the Food and Drug Administration approved the first genetic treatments for sickle cell disease. So far, only a few patients have received the long-awaited treatments.
