STAT+: FDA offers new details on quicker approval pathway for personalized gene therapies
Rare disease advocates applauded the FDA's move to speed access to treatments.
The Food and Drug Administration (FDA) has released draft guidance that aims to expedite the approval process for personalized gene therapies, a development that is being welcomed by rare disease advocates. This move comes as breakthroughs in gene therapies have captured the attention of researchers and pharmaceutical companies, who believe these personalized medicines could significantly benefit patients with rare mutations.
Paul Scribner, vice president of community programs and education at the PKD Foundation, highlighted the significance of the FDA's initiative at a recent rare disease event.
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