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| http://dx.doi.org/10.5152/tud.2014.9513569 | DOI Listing |
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Late-Onset Rheumatoid Arthritis (LORA): A Diagnostic and Therapeutic Challenge Among Older Patients Visiting a Poorly Resourced Health-Care Setting.
Mediterr J Rheumatol
December 2024
Department of Internal Medicine, B. P. Koirala Institute of Health Sciences, Dharan, Nepal.
Late-onset rheumatoid arthritis (LORA) presents a unique diagnostic challenge among older patients, particularly in poorly resourced healthcare settings. As global life expectancy increases, so does the prevalence of LORA, a condition that differs significantly from young-onset rheumatoid arthritis (YORA). This review explores the distinct clinical presentation, differential diagnosis, laboratory findings, and treatment challenges of LORA, emphasising its impact on low- and middle-income countries.
View Article and Find Full Text PDFCharacterization of children with early onset pediatric multiple sclerosis.
Eur J Paediatr Neurol
January 2025
Department of Pediatric Neurology, Children's Hospital Datteln, Witten/Herdecke University, Datteln, Germany. Electronic address:
Background: Early onset pediatric multiple sclerosis (EOPMS) provides an early window of opportunity to understand the mechanisms leading to MS.
Objective: To investigate clinical, laboratory and imaging differences between children with early onset pediatric MS (<11 years, EOPMS) and late onset pediatric MS (≥11 years, LOPMS).
Methods: Mostly prospectively collected data of children with MS including clinical presentation, MRI at onset, time to second relapse, relapse rate, treatment history, and CSF markers were eligible.
Management of presymptomatic juvenile patients with late-onset Pompe disease (LOPD).
Neuromuscul Disord
January 2025
ERN-NMD Center for Neuromuscular Disorders of Messina - Department of Clinical and Experimental Medicine, University of Messina, Messina, Italy. Electronic address:
Late-onset Pompe disease (LOPD) includes patients from 1 year of age to adulthood. The vast heterogeneity in clinical manifestations and disease progression is not fully explained; however, a short disease duration and a young age seem to be good predictors of a better response to treatment. For this purpose, we investigated and followed up a cohort of 13 juvenile patients with LOPD from the clinical and therapeutic point of view, mainly pointing out the transition from presymptomatic to symptomatic status.
View Article and Find Full Text PDFLate-Onset Krabbe Disease: Case Report of Two Patients in a Chinese Family and Literature Review.
Mol Genet Genomic Med
February 2025
Department of Orthopeadic Surgery, Sun Yat-Sen Memorial Hospital, Sun Yat-Sen University, Guangzhou, People's Republic of China.
Background: Krabbe disease (KD; globoid cell leucodystrophy) is a rare autosomal recessive lipid storage disorder that affects the white matter of the peripheral and central nervous. Late-onset KD is less frequently diagnosed and often presents with milder symptoms, making accurate diagnosis challenging, especially when distinguishing it from peripheral neuropathy. In this report, we present two cases of late-onset KD in a Chinese family.
View Article and Find Full Text PDFJoint TITE-CRM: A Design for Dose Finding Studies for Therapies with Late-Onset Safety and Activity Outcomes.
Stat Biopharm Res
March 2024
MRC Biostatistics Unit, University of Cambridge, Cambridge, UK.
In Phase I/II dose-finding trials, the objective is to find the Optimal Biological Dose (OBD), a dose that is both safe and shows sufficient activity that maximizes some optimality criterion based on safety and activity. In cancer, treatment is typically given over several cycles, complicating the identification of the OBD as both toxicity and activity outcomes may occur at any point throughout the follow up of multiple cycles. In this work we present and assess the Joint TITE-CRM, a model-based design for late onset toxicities and activity based on the well-known TITE-CRM.
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