Orphan medical devices: addressing the regulatory and access gaps in the EU and US. [PDF]
Akodad S, Haon B, Rochaix L, Stevens H.
europepmc +1 more source
ABSTRACT Objective This study aims to identify both fluid and neuroimaging biomarkers for CSF1R‐RD that can inform the optimal timing of treatment administration to maximize therapeutic benefit, while also providing sensitive quantitative measurements to monitor disease progression.
Tomasz Chmiela +13 more
wiley +1 more source
Guideline in Action: General Supportive Early Management: Blood Pressure and Glucose. [PDF]
Wang J, Tahsili-Fahadan P.
europepmc +1 more source
PHP152 The Early Benefit Assessment of Drugs That are Launched Before 2011 [PDF]
openaire +1 more source
Changes in Immune‐Inflammation Status and Acute Ischemic Stroke Prognosis in Prospective Cohort
ABSTRACT Background Inflammation is a critical risk factor for poor outcomes in cerebral infarction. Prior studies focused primarily on baseline inflammation status, neglecting dynamic longitudinal changes. We try to investigate the association between immune‐inflammation status alterations and stroke prognosis, and evaluated three systemic biomarkers'
Songfang Chen +11 more
wiley +1 more source
A review of evidence supporting amyloid beta reduction as a surrogate endpoint in Alzheimer's disease. [PDF]
Chen T +13 more
europepmc +1 more source
Chronological and Spatial Distribution of Skeletal Muscle Fat Replacement in FHL1‐Related Myopathies
ABSTRACT Objectives Variants in the FHL1 gene cause FHL1‐related myopathies (FHL1‐RMs), a group of neuromuscular disorders with diverse clinical presentations. This study aimed to comprehensively characterize the spatial and temporal patterns of skeletal muscle fat replacement throughout the whole body in FHL1‐RMs, to examine disease progression over ...
Rui Shimazaki +8 more
wiley +1 more source
Quality of life in oncology drug approvals: evidence from Swiss decisions in relation to global regulatory trends. [PDF]
Horst A +3 more
europepmc +1 more source
Use of Symptomatic Drug Treatment for Fatigue in Multiple Sclerosis and Patterns of Work Loss
ABSTRACT Objective To describe the use of central stimulants and amantadine for fatigue in MS and evaluate a potential association with reduced work loss in people with MS. Methods We conducted a nationwide, matched, register‐based cohort study in Sweden (2006 to 2023) using national registers with prospective data collection.
Simon Englund +3 more
wiley +1 more source
ABSTRACT Objective This study aimed to systematically observe the clinical manifestations, immune cell subsets, and dynamic changes in serological indicators in patients with myasthenia gravis (MG) before and after efgartigimod (EFG) treatment. Methods We analyzed the baseline data, laboratory parameters, and lymphocyte subset proportions in MG ...
Tiancheng Luo +9 more
wiley +1 more source

