The treatment, developed by Regeneron Pharmaceuticals, is for a very rare form of deafness. But it represents a medical milestone.
gene therapy
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Gene therapy for a rare type of deafness shows lasting results
Researchers say a gene therapy allowed deaf children and adults as old as 32 to hear for the first time. The benefits have persisted for more than two years for some patients.
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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.
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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.
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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.
