Elevidys, a gene therapy developed to combat DMD, received accelerated approval in mid-2023.[50] While it fell short of achieving its primary Phase III endpoint,[51] subsequent follow-up studies showed stable disease among recipients and some improvements in other measures of motor function.[52] Most recently, the FDA has narrowed the indication for Elevidys to ambulatory DMD patients who are at least 4 years old and issued a black box warning to highlight the risk of serious liver injury and failure.[53] Similarly, two- to five-year outcome studies of infants receiving gene therapy for spinal muscular atrophy (SMA)a rare, inherited neuromuscular disorder characterized by the progressive loss of motor neurons in the spinal cord and brainstemsuggest significant functional improvements, although about half of patients experienced a treatment-emergent adverse effect.[54, 53] Data for use of CAR-T therapies to treat blood cancers, including B-cell lymphomas and multiple myelomas, indicates that a majority of recipients achieve durable long-term remission, far more than with previous therapies.[55] There is longer-term follow-up evidence for CAR-T therapies for these diseases
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doi: 10.1046/j.1365-201x.1999.00618.x
