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
Untargeted LC-MS/MS Metabolite Profiling of Wild, Field-Domesticated and Tissue-Cultured Rhizomes of the Endangered Medicinal Fern <i>Cibotium barometz</i>. [PDF]
Su Y, Qin G, Zhou C, Guan D, Song J.
europepmc +1 more source
Early Clinical, Imaging, and Pathological Characteristics of SRPK3/TTN‐Digenic Myopathy
ABSTRACT Objective SRPK3/TTN‐digenic myopathy was recently established as a skeletal muscle myopathy caused by digenic inheritance. This study characterizes the early clinical presentation of SRPK3/TTN‐digenic myopathy in one previously reported and seven newly identified pediatric patients.
Rotem Orbach +23 more
wiley +1 more source
Surgery and positive Bakry-Émery Ricci curvature. [PDF]
Reiser P, Tripaldi F.
europepmc +1 more source
ABSTRACT Introduction Glucagon‐like peptide‐1 receptor agonists (GLP‐1 RAs) have demonstrated significant weight‐reducing effects and may offer benefits in idiopathic intracranial hypertension (IIH); however, recent concerns about the risk of non‐arteritic anterior ischemic optic neuropathy (NAION) have emerged.
Faisal A. Al‐Harbi +9 more
wiley +1 more source
Adaptive Weighting-Based Multi-Station Doppler Orbit Determination for Noncooperative LEO Satellites. [PDF]
Lei M, Liu Y, Yang Z, Li H, Fang Z.
europepmc +1 more source
Sertraline Treatment Can Mimic Niemann‐Pick Type C Biomarker Profile: A Diagnostic Pitfall
ABSTRACT Background Oxysterols (cholestane‐3β,5α,6β‐triol and 7‐ketocholesterol) and N‐palmitoyl‐O‐phosphocholineserine (PPCS) are sensitive biomarkers for Niemann‐Pick disease type C (NPC) screening. However, false‐positive results occur, with a biomarker profile suggestive of NPC despite the absence of pathogenic variants in genes involved in NPC or ...
Maria Makrygianni +19 more
wiley +1 more source
Proposal of a procedure to stratify the reidentification risk of medical data: RIMEDA. [PDF]
Behre S +3 more
europepmc +1 more source
Neuromorphic weighted sum with magnetic skyrmions
Magnetic skyrmions are topological magnetic solitons that can be stabilized at room temperature in magnetic thin films or heterostructures with optimized magnetic anisotropy and Dzyaloshinskii-Moriya interaction. Recent experimental studies have shown, often separately, that magnetic skyrmions can be nucleated [1-4], moved [2,4], annihilated [3] and ...
da Câmara Santa Clara Gomes, Tristan +12 more
openaire +6 more sources
Comparative Effectiveness and Safety of Inebilizumab Versus Rituximab in AQP4‐IgG‐Positive NMOSD
ABSTRACT Objective Rituximab (anti‐CD20, RTX) and inebilizumab (anti‐CD19, INE) represent B‐cell‐depleting therapies used for aquaporin‐4 antibody‐positive (AQP4‐IgG+) neuromyelitis optica spectrum disorder (NMOSD); however, direct comparative evidence remains limited.
Jie Lin +11 more
wiley +1 more source

