Small molecules in idiopathic inflammatory myopathies: a systematic review and a multicenter case series about Janus kinase inhibitors and apremilast.

Objective: Idiopathic inflammatory myopathies (IIM) are rare autoimmune diseases that primarily affect striated muscles; skin, joints, and lungs may be involved with different degrees of severity. Traditional treatment relies on high-dose glucocorticoids and conventional synthetic disease-modifying antirheumatic drugs.

Methods: A growing amount of evidence is demonstrating the potential role of novel treatments in the management of IIM.

Burden of RSV infections among young children in primary care: a prospective cohort study in five European countries (2021-23).

Background: The majority of respiratory syncytial virus (RSV) infections in young children are managed in primary care, however, the disease burden in this setting remains poorly defined.

Methods: We did a prospective cohort study in primary care settings in Belgium, Italy, Spain, the Netherlands, and the UK during the RSV seasons of 2020-21 (UK only; from Jan 1, 2021), 2021-22, and 2022-23. Children aged younger than 5 years presenting to their general practitioner or primary care paediatrician with symptoms of an acute respiratory tract infection were eligible for RSV testing.

Cord blood platelet-rich plasma: proteomics analysis for ophthalmic applications.

Our objective is to determine the protein and complements constituents of Cord blood Platelet-rich plasma (CB-PRP), based on the hypothesis that it contains beneficial components capable of arresting or potentially decelerating the advancement of atrophic age-related macular degeneration (dry-AMD), with the support of radiomics. Two distinct pools of CB-PRP were assessed, each pool obtained from a total of 15 umbilical cord-blood donors. One aliquot of each pool respectively was subjected to proteomic analysis in order to enhance the significance of our findings, by identifying proteins that are shared between the two sample pools and gaining insights into the pathways they are associated with.

Widening the infantile hypotonia with psychomotor retardation and characteristic Facies-1 Syndrome's clinical and molecular spectrum through NALCN structural analysis.

Introduction: Infantile hypotonia with psychomotor retardation and characteristic facies-1 (IHPRF1, MIM#615419) is a rare, birth onset, autosomal recessive disorder caused by homozygous or compound heterozygous truncating variants in gene (MIM#611549) resulting in a loss-of-function effect.

Methods: We enrolled a new IHPRF1 patients' cohort in the framework of an international multicentric collaboration study. Using specialized pathogenicity predictors and structural analyses, we assessed the mechanistic consequences of the deleterious variants retrieved on NALCN structure and function.

Regression of the Flow Signal from the Neovascular Network in AMD Neovascular Membranes Treated with Faricimab.

Objectives: To report the occurrence of the regression of the flow signal from the neovascular network in macular neovascularizations (MNVs), developing in the context of age-related macular degeneration (AMD), treated with faricimab in a treat-and-extend regimen.

Methods: Eyes affected by AMD-related MNV and treated with faricimab intravitreal injections in a treat-and-extend (TE) regimen were consecutively retrospectively screened in five specialized retina centers. Changes in neovascular network characteristics during the course of the treatment were analyzed.