ADAPT NXT: Fixed Cycles or Every‐Other‐Week IV Efgartigimod in Generalized Myasthenia Gravis
ABSTRACT Objective This phase 3b, open‐label, randomized ADAPT NXT study investigated the efficacy, safety, and tolerability of efgartigimod administered in either a fixed cycles dosing regimen (3 cycles of 4 once‐weekly infusions, with 4 weeks between cycles) or a cycle followed by every‐other‐week (Q2W) dosing.
Ali A. Habib+16 more
wiley +1 more source
Objectives: In this study, we evaluated the correlation between severity of deformity and cardiopulmonary function with regards to echocardiographic and spirometric findings.
Alper Hazım Gürsu+4 more
doaj +1 more source
KIF5A p.Pro986Leu Risk Variant and Accelerated Progression of Amyotrophic Lateral Sclerosis
ABSTRACT This study explored the impact of KIF5A rs113247976 (p.Pro986Leu), a risk allele for amyotrophic lateral sclerosis (ALS), on phenotypic variability in two Italian ALS cohorts (discovery, n = 865; replication, n = 1174). The minor allele (T) frequency was 0.015.
Arianna Manini+24 more
wiley +1 more source
Interstitial lung disease in Malta [PDF]
Aim: To establish the prevalence, management and response to treatment of interstitial lung disease (ILD) in Malta. Methodology: The personal files of 102 living and 26 deceased patients with ILD under the care of 4 respiratory physicians were reviewed ...
Fenech, Manwel+2 more
core
Validity and Reliability of Clinical and Patient‐Reported Outcomes in Multisystem Proteinopathy 1
ABSTRACT Objective Valosin‐containing protein (VCP)‐associated multisystem proteinopathy 1 (MSP1) is caused by variants in the VCP gene. MSP1 results in various phenotypes including progressive myopathy, Paget's disease of bone, frontotemporal dementia, amyotrophic lateral sclerosis, and parkinsonism, among others.
Lindsay N. Alfano+15 more
wiley +1 more source
Is there an association between postural balance and pulmonary function in adults with asthma?
OBJECTIVE: Asthma may cause systemic repercussions due to its severity and the effects of treatment. Our objective was to compare posture, balance, functional capacity, and quality of life (QOL) according to the severity of disease, as assessed by ...
Vivian Pinto de Almeida+5 more
doaj +1 more source
Respiratory and cardiovascular function at rest and during exercise testing in a healthy working population: effects of outdoor traffic air pollution [PDF]
P Volpino+9 more
openalex +1 more source
DFV890: a new oral NLRP3 inhibitor—tested in an early phase 2a randomised clinical trial in patients with COVID-19 pneumonia and impaired respiratory function [PDF]
Ildikó Madurka+99 more
openalex +1 more source
Translating Muscle RNAseq Into the Clinic for the Diagnosis of Muscle Diseases
ABSTRACT Objective Approximately half of patients with hereditary myopathies remain without a definitive genetic diagnosis after DNA next‐generation sequencing (NGS). Here, we implemented transcriptome analysis of muscle biopsies as a complementary diagnostic tool for patients with muscle disease but no definitive genetic diagnosis after exome ...
Alba Segarra‐Casas+24 more
wiley +1 more source
Can 1st and 6th month pulmonary function test follow-ups give an idea about the long-term respiratory effects of COVID-19 pneumonia? [PDF]
Serkan Emre Eroğlu+7 more
openalex +1 more source