Myostatin/Smad3 in Spinal Muscular Atrophy: Biomarkers of Disease Severity and Treatment Response
ABSTRACT Objective Given the role of Myostatin (MSTN) in muscle homeostasis and its modulation by current spinal muscular atrophy (SMA) therapies, we investigated serum MSTN and, for the first time, its downstream effector Smad3 as candidate non‐invasive biomarkers in SMA patients undergoing disease‐modifying therapies (DMTs).
Giorgia Riolo +11 more
wiley +1 more source
Management and outcomes of patients undergoing high-risk oncologic pancreatic surgery: do patients presented at pancreatic multidisciplinary tumor board fare better? [PDF]
Breit C +6 more
europepmc +1 more source
ABSTRACT Objective Febrile seizures (FS) are currently classified as simple or complex primarily to describe seizure characteristics, with limited ability to identify the underlying etiology. We evaluated whether molecular diagnosis could complement conventional classification and improve the diagnosis and management of FS.
Jia Wang +22 more
wiley +1 more source
EXCEL-Punjabi: the process of using a community based participatory research approach for a culturally-tailored exercise oncology intervention. [PDF]
Bansal M +8 more
europepmc +1 more source
Cerebrospinal Fluid GAD65‐Antibody Levels Accurately Identify Relevant Neurological Diseases
ABSTRACT Retrospective cohort study to investigate which factors most reliably distinguish incidental GAD65‐antibodies (iGAD65) from true GAD65‐antibody neurological disease (tGAD65). 167 adults with positive serum and CSF GAD65‐antibodies (> 0.02 nmol/L by radioimmunoassay) were classified as tGAD65 or iGAD65, with diagnostic performance of ...
Max Charles Herman +9 more
wiley +1 more source
Prevalence of ties to industry-funded organizations among patient and consumer representatives on FDA advisory committees. [PDF]
Kanter GP, Mankowitz T.
europepmc +1 more source
Systemic sclerosis (SSc) is a rare autoimmune disease defined by immune dysregulation, vasculopathy, and progressive fibrosis of the skin and internal organs. Despite advances in care, major complications such as interstitial lung disease (ILD) and myocardial involvement remain the leading causes of morbidity and mortality.
Cristiana Sieiro Santos +2 more
wiley +1 more source
Managing multimorbidity in severe asthma: comparison of resources and perspectives between severe asthma specialists and general respiratory physicians across Europe. [PDF]
Matīsa D +65 more
europepmc +1 more source
Discordance Between Patient and Physician Global Assessments in Early Systemic Sclerosis
Objective This study aims to identify factors associated with patient global assessment (PtGA) and physician global assessment (PhGA) and discordance between them in systemic sclerosis (SSc). Methods Data from adults with early SSc (<5 years) from the Collaborative National Quality and Efficacy Registry were included.
Ellen Romich +35 more
wiley +1 more source
Objective To investigate the association between rheumatoid arthritis (RA) and coronary artery calcium (CAC) prevalence, incidence, and progression over four years in adults without prior cardiovascular disease. Methods A case‐cohort study within the Brazilian Longitudinal Study of Adult Health (ELSA‐Brasil) included 585 participants (86 patients with ...
Patrícia Fonseca Estrada +7 more
wiley +1 more source

