Inherited Retinal Degenerations and Non-Neovascular Age-Related Macular Degeneration: Progress and Unmet Needs.

Inherited retinal degeneration (IRD) disease and age-related macular degeneration (AMD) are leading causes of irreversible vision loss and blindness. Although significant progress has advanced the field in the past 5 years, significant challenges remain. The current article reviews the accomplishments and research advances that have fueled the development of treatments for patients with IRD and AMD, including the first approved gene-augmentation treatment for RPE65-related retinal degeneration and complement inhibition therapies to slow progression of geographic atrophy (GA) in AMD.

Informing Endpoints for Clinical Trials of Geographic Atrophy.

Geographic atrophy (GA), the non-neovascular advanced form of age-related macular degeneration, remains an important disease area in which treatment needs are currently unmet. Recent clinical trials using drugs that target the complement pathway have shown modest yet consistent reductions in GA expansion but without commensurate changes in measures of visual function. In this review, we summarize information from the wide range of studies describing the characteristics of GA morphology and enumerate the factors influencing the growth rates of lesions and the directionality of expansion.

"I believe it will not get worse": A mixed-methods longitudinal study about patient's perspective of recently developed patellofemoral pain.

Objectives: This study aimed to: (i) understand how women perceived their recently developed patellofemoral pain (PFP) regarding its cause, prognosis, and willingness to seek treatment; (ii) investigate self-reported function, knee-related quality of life (QoL), fear of movement, and physical activity level at the onset of PFP.

Design: Mixed-methods longitudinal study.

Setting: University.

Long-term natural history of ellipsoid zone width in -retinopathy.

Aims: To investigate the long-term natural history of ellipsoid zone (EZ) width in -retinopathy.

Methods: EZ width measurements from optical coherence tomography were retrospectively obtained from 110 eyes of 55 participants with molecularly confirmed biallelic -retinopathy. We used a hierarchical Bayesian method to construct and compare different mathematical models describing the long-term decline of EZ width.