Results 51 to 60 of about 834,867 (353)

Effectiveness and Safety of Nusinersen and Risdiplam in Spinal Muscular Atrophy: A Systematic Review

open access: yesAnnals of Clinical and Translational Neurology, EarlyView.
ABSTRACT Objective Spinal Muscular Atrophy (SMA) is a rare genetic disorder marked by progressive muscle weakness and mobility loss. It has a profound physical, emotional and social impact on patients and caregivers, requiring comprehensive medical and supportive care.
Amin Mehrabian   +9 more
wiley   +1 more source

Effectiveness and safety of baricitinib for juvenile idiopathic arthritis associated uveitis or chronic anterior antinuclear antibody positive uveitis

open access: yesArthritis Care &Research, Accepted Article.
Objectives Evaluate the efficacy and safety of baricitinib in paediatric patients with active JIA‐U or chronic anterior ANA‐positive uveitis, who had an inadequate response to MTX or bDMARDs. Methods JUVE‐BRIGHT was an open‐label, active‐controlled, Phase‐3 multicentre trial which utilized a novel design, including 1:1 randomization to an active ...
Athimalaipet V. Ramanan   +7 more
wiley   +1 more source

Respiratory Tract Infections and the Role of Biologically Active Polysaccharides in Their Management and Prevention

open access: yesNutrients, 2017
Respiratory tract infections (RTIs) are the most common form of infections in every age category. Recurrent respiratory tract infections (RRTIs), a specific form of RTIs, represent a typical and common problem associated with early childhood, causing ...
M. Jeseňák   +2 more
semanticscholar   +1 more source

Clinical Conditions Associated With a High Antinuclear Antibody Titer in Individuals Without Autoimmune Disease

open access: yesArthritis Care &Research, EarlyView.
Objective Antinuclear antibodies (ANAs) are present at high titers in 2% of the general population, but their clinical significance in individuals without an autoimmune (AI) disease is not known. We tested the hypothesis that the presence of a high ANA titer in non‐AI conditions is associated with disease.
Matthew Chung   +7 more
wiley   +1 more source

Sarilumab in Polyarticular‐Course Juvenile Idiopathic Arthritis: Dose‐Finding and One‐Year Analysis of a Phase 2b, Open‐Label, Multicenter Study

open access: yesArthritis Care &Research, Accepted Article.
Objective This study assessed sarilumab in treating patients with polyarticular‐course juvenile idiopathic arthritis (pcJIA). Methods This phase 2b, open‐label study (NCT02776735) consisted of three sequential parts (each with a core‐treatment and extension‐phase). During part 1, three doses were assessed in two weight groups (Group A/B: ≥30–60 kg/≥10–<
Fabrizio De Benedetti   +19 more
wiley   +1 more source

Coronavirus Infections in the Central Nervous System and Respiratory Tract Show Distinct Features in Hospitalized Children

open access: yesIntervirology, 2017
Background/Aims: Coronavirus (CoV) infections induce respiratory tract illnesses and central nervous system (CNS) diseases. We aimed to explore the cytokine expression profiles in hospitalized children with CoV-CNS and CoV-respiratory tract infections ...
Yuanyuan Li   +13 more
semanticscholar   +1 more source

Pharmacokinetics, Pharmacodynamics, and Safety of Subcutaneous Belimumab in Pediatric Patients with Systemic Lupus Erythematosus: A Multicenter, Open‐Label Trial

open access: yesArthritis Care &Research, Accepted Article.
Objective This study aimed to characterize the pharmacokinetics, pharmacodynamics, safety, and exploratory efficacy of subcutaneous belimumab in pediatric patients with active systemic lupus erythematosus (SLE) receiving standard therapy. Methods This single‐arm, multicenter, open‐label trial (GSK study 200908; NCT04179032) used three‐weight‐band ...
Hermine I. Brunner   +14 more
wiley   +1 more source

Viral respiratory tract infections in young children with cystic fibrosis: a prospective full-year seasonal study

open access: yesVirology Journal, 2019
Background Viral respiratory tract infections are common during early childhood. How they impact cystic fibrosis lung disease history in young children is poorly known.
Mathilde Eymery   +6 more
doaj   +1 more source

Generation of an Induced Pluripotent Stem Cell‐Derived Alveolar Type II In Vitro Model to Study Influenza A Virus Infection and Drug Treatments

open access: yesAdvanced Healthcare Materials, EarlyView.
Patient‐specific induced pluripotent stem cells (iPSCs) can be differentiated into alveolar type II cells (iAT2s), expanded as 3D alveolospheres, and grown at physiologically relevant air–liquid interface (ALI). This study shows for the first time the infectability of iAT2s by the influenza A virus (IAV) and proves their responsiveness to the well ...
Lena Gauthier   +7 more
wiley   +1 more source

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