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.
rare diseases
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
For kids with rare genetic disorders, customized CRISPR treatments offer hope
The gene-editing technique is effective for treating some illnesses but it’s been too expensive to consider it for rare conditions. A new approach in the works could make it more widely available.
