Gene therapy: Staying grounded

By Jose Alain Sahel Jr., MD, FARVO
and Mark Pennesi, MD, PhD

Since the Food and Drug Administration (FDA) approved voretigene neparvovec-rzyl (Luxturna™) from Spark Therapeutics roughly one year ago, patients with inherited retinal dystrophies (IRDs) have been given reason to believe they may see light at the end of their darkening prognosis. Two clinician scientists studying IRDs offer their perspectives on the scientific and clinical challenges that still face their field and patients. 

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Looking toward the next 50 years

Gene therapy: Staying grounded

Since the Food and Drug Administration (FDA) approved voretigene neparvovec-rzyl (LuxturnaTM) from Spark Therapeutics roughly one year ago, patients with inherited retinal dystrophies (IRDs) have been given reason to believe they may see light at the end of their darkening prognosis.

ARVO members among 2018 Champalimaud Vision Award winners

Seven scientists recognized for the first successful gene therapy to cure an inherited human disease