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
Assessment of safety culture in adults and paediatric critical care units in governmental hospitals in Kuwait: a cross-sectional study. [PDF]
Alsaleh F +3 more
europepmc +1 more source
Paediatric Nasogastric Tube Insertion Practices Across Australia and New Zealand: A Survey of Clinicians. [PDF]
Liu AKS +5 more
europepmc +1 more source
Movement Disorders in Aicardi–Goutières Syndrome and Response to Immunomodulation
ABSTRACT This study characterizes movement disorders and treatment responses in seven children with Aicardi–Goutières syndrome (AGS). We retrospectively evaluated motor phenotypes, neuroimaging, and interferon signatures in patients treated with baricitinib or anifrolumab. Spasticity affected all patients, while dystonia was present in 4/7.
Enrique Gonzalez Saez‐Diez +10 more
wiley +1 more source
Integrating early clinical exposure, digital simulation, and soft-skill development into undergraduate paediatric dentistry curricula. [PDF]
Hisham ARB.
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
Fosfomycin combination therapy in high-risk paediatric patients with multidrug-resistant infections: a retrospective study. [PDF]
Denina M +7 more
europepmc +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
Being There: Danish Nurses' Lived Experiences of Companionship With Families in Home-Based Paediatric Palliative Care in Regional Settings. [PDF]
Alm A, Beck M.
europepmc +1 more source

