The burden of X-linked retinitis pigmentosa (XLRP) on patient experience and patient-reported outcomes (PROs): findings from the EXPLORE XLRP-2 study.

Background/aims: X-linked retinitis pigmentosa (XLRP) is considered one of the most severe forms of retinitis pigmentosa (RP), accounting for 5-15% of all RP cases and primarily affecting males. However, the real-world humanistic impacts of this disease on patients are poorly investigated, especially with respect to burdens faced by patients with varying disease severities.

Methods: EXPLORE XLRP-2 was an exploratory, multicentre, non-interventional study.

Expanding the phenotype of : a range of macular dystrophies as the major clinical manifestations in patients with a dominant splicing variant.

Inherited retinal dystrophies (IRDs) are a clinically and genetically heterogeneous group of disorders that often severely impair vision. Some patients manifest poor central vision as the first symptom due to cone-dysfunction, which is consistent with cone dystrophy (COD), Stargardt disease (STGD), or macular dystrophy (MD) among others. Here, we aimed to identify the genetic cause of autosomal dominant COD in one family.

Adverse events of antibody-drug conjugates on the ocular surface in cancer therapy.

Antibody-drug conjugates consist of a monoclonal antibody attached to a cytotoxic therapeutic molecule by a connector. This association allows a highly specific therapy, which increases their effectiveness and decreases their potential toxicity. This new therapy emerged approximately 20 years ago; since then, numerous combinations have appeared in the field of treatment-related neoplasms as an alternative for patients who do not achieve good results with conventional treatment options.