Publications by authors named "Adam A DeBusk"
Article Synopsis
- Lenadogene nolparvovec is a novel gene therapy showing significant promise for improving visual acuity in patients with Leber hereditary optic neuropathy (LHON) who have the m.11778G>A ND4 mutation, with updated data increasing the number of treated patients from 76 to 174.
- The therapy was compared against an external control group and resulted in a mean improvement in visual acuity of -0.30 logMAR, which is equivalent to +15 ETDRS letters, showing clinically relevant and sustained benefits over a follow-up period of up to 3.9 years.
- Results indicate that most treated eyes retained measurable vision compared to less than half of those in the natural history group, and a
Background: Patients with typical features of pseudotumor cerebri syndrome (PTCS) must undergo lumbar puncture (LP) to demonstrate elevated opening pressure and cerebrospinal fluid (CSF) analysis to rule out alternative diagnoses. As LP may be associated with significant morbidity, this study aims to determine its necessity in diagnosing typical PTCS.
Methods: Retrospective chart review at 3 university-based neuro-ophthalmology practices included women aged 18-45 years with body mass index >25, papilledema, negative neuroimaging, and who met criteria for PTCS or probable PTCS.
Objective: This report presents a cross-sectional analysis of the baseline characteristics of subjects with Leber hereditary optic neuropathy enrolled in the gene therapy trials RESCUE and REVERSE, to illustrate the evolution of visual parameters over the first year after vision loss.
Methods: RESCUE and REVERSE were 2 phase III clinical trials designed to assess the efficacy of rAAV2/2-ND4 gene therapy in ND4-LHON subjects. At enrollment, subjects had vision loss for ≤6 months in RESCUE, and between 6 and 12 months in REVERSE.
This work aimed to compare the evolution of visual outcomes in Leber hereditary optic neuropathy (LHON) patients treated with intravitreal gene therapy to the spontaneous evolution in prior natural history (NH) studies. A combined analysis of two phase three randomized, double-masked, sham-controlled studies (REVERSE and RESCUE) and their joint long-term extension trial (CLIN06) evaluated the efficacy of rAAV2/2- vs. 11 pooled NH studies used as an external control.
View Article and Find Full Text PDFPurpose: To evaluate the efficacy of a single intravitreal injection of rAAV2/2-ND4 in subjects with visual loss from Leber hereditary optic neuropathy (LHON).
Design: RESCUE is a multicenter, randomized, double-masked, sham-controlled, phase 3 clinical trial.
Participants: Subjects with the m.