rare diseases
Where patients live matters for access to gene therapy
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.
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.
A promising genetic treatment tailor-made for a baby born with a rare disorder
For the first time, doctors have created a customized treatment using the revolutionary gene-editing technique known as CRISPR to treat a baby with a rare, life-threatening genetic disorder.
