During the coronavirus disease 2019 (COVID-19) pandemic, outpatient clinics were shut down to stem virus spread. Before COVID-19, Henry Ford Health System (Detroit, Michigan) used telemedicine within limited subsets of patients. After Henry Ford Health System clinics closed on March 17, 2020, the need to continue care necessitated a rapid adaptation of telemedicine. This presented an opportunity for widespread telemedicine use within the multidisciplinary head and neck oncology team. The purpose of this article is to describe the associations between patient demographic characteristics and socioeconomic disparities and the engagement in telemedicine during the pandemic.
Summary
A first-in-human clinical trial of gene therapy in Leber congenital amaurosis due to mutations in the
GUCY2D
gene is underway, and early results are summarized. A recombinant adeno-associated virus serotype 5 (rAAV5) vector carrying the human
GUCY2D
gene was delivered by subretinal injection to one eye in three adult patients with severe visual loss, nystagmus, but preserved retinal structure. Safety and efficacy parameters were monitored for 9 months post-operatively. No systemic toxicity was detected; there were no serious adverse events, and ocular adverse events resolved. P1 and P2 showed statistically significant rod photoreceptor vision improvement by full-field stimulus testing in the treated eye. P1 also showed improvement in pupillary responses. Visual acuity remained stable from baseline in P1 and P2. P3, however, showed a gain of 0.3 logMAR in the treated eye, indicating greater cone-photoreceptor function. The results show safety and both rod- and cone-mediated efficacy of this therapy.
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