Results 71 to 80 of about 277,897 (298)
Safety and Tolerability of Givinostat: Evidence From Real‐World and Clinical Practice
ABSTRACT Objective The aim of our study was to establish the prevalence of adverse events in a real‐world setting in boys living with Duchenne muscular dystrophy (DMD) treated with givinostat as part of an Expanded Access Program (EAP) in Italy. Methods The cohort included 90 ambulant boys, with age when treatment started between 6 and 23 years (mean ...
Marika Pane +19 more
wiley +1 more source
ABSTRACT Chimeric antigen receptor (CAR) T‐cell therapy has been investigated in neurological diseases, encompassing both central nervous system malignancies and autoimmune disorders, thereby extending its application beyond hematological cancers.
Omar Alqaisi +5 more
wiley +1 more source
Whole-genome sequencing and the clinician: a tale of two cities [PDF]
Clinicians are faced with unprecedented opportunities to identify the genetic aetiologies of hitherto molecularly uncharacterised conditions via the use of high-throughput sequencing.
A. R. Foley +21 more
core +2 more sources
Objective Sjögren's disease is an autoimmune disorder that can impact multiple organ systems, including the peripheral nervous system (PNS). PNS manifestations, which can exist concurrently, include mononeuropathies, polyneuropathies, and autonomic nervous system neuropathies.
Anahita Deboo +88 more
wiley +1 more source
Flux control of cytochrome c oxidase in human skeletal muscle [PDF]
In the present work, by titrating cytochrome c oxidase (COX) with the specific inhibitor KCN, the flux control coefficient and the metabolic reserve capacity of COX have been determined in human saponin-permeabilized muscle fibers. In the presence of the
Attardi, Giuseppe +5 more
core +1 more source
Immune Checkpoint Inhibitor–Related Myositis and Associated Triad Overlap Syndrome
Objective Immune checkpoint inhibitor (ICI) myositis is a rare but a highly morbid condition, particularly with the ICI myositis triad syndrome of myositis, myocarditis, and myasthenia gravis. We report the clinical characteristics of ICI myositis and all‐cause mortality in these patients.
Selene Rubino +9 more
wiley +1 more source
Background: Juvenile idiopathic inflammatory myopathies (JIIM) are rare and heterogeneous. Subtype identification is important for treatment. Materials and Methods: Patients below 18 years diagnosed as idiopathic inflammatory myopathy (IIM) according to ...
Sundaram Challa +7 more
doaj +1 more source
Rheumatologic Manifestations of Patients With Type B Insulin Resistance
Objective The objectives of this study were to identify laboratory and clinical features associated with type B insulin resistance (TBIR), a rare condition caused by autoantibodies that inhibit the insulin receptor, most frequently occurring in the setting of systemic lupus erythematosus (SLE), and to increase awareness of this rare, life‐threatening ...
S. Amara Ogbonnaya +4 more
wiley +1 more source
Specific Preferences in Lineage Choice and Phenotypic Plasticity of Glioma Stem Cells Under BMP4 and Noggin Influence [PDF]
Although BMP4-induced differentiation of glioma stem cells (GSCs) is well recognized, details of the cellular responses triggered by this morphogen are still poorly defined.
Arakaki, Naomi +10 more
core +1 more source
Characteristics and outcomes of male participants in the Australian Scleroderma Cohort Study (ASCS).
Objective To determine the differences in demographic, serologic and clinical characteristics between male and female patients with systemic sclerosis (SSc) in an Australian cohort. Methods Retrospective observational study using data from the Australian Scleroderma Cohort Study (ASCS). Males (M) were compared to females (F) using chi‐squared, Wilcoxon
Emily Lin +14 more
wiley +1 more source

