Results 141 to 150 of about 586,644 (327)
ABSTRACT Objective Facioscapulohumeral muscular dystrophy type 1 (FSHD1) is a progressive neuromuscular disorder with no approved treatments. Identifying reliable biomarkers is critical to monitor disease severity, activity, and progression. Interleukin‐6 (IL‐6) has been proposed as a candidate biomarker, but longitudinal validation is limited ...
Jonathan Pini +13 more
wiley +1 more source
Manabu Akazawa,1 Ataru Igarashi,2 Nozomi Ebata,3 Tatsunori Murata,4 Shigeki Zeniya,4 Yuri Haga,5 Kazutaka Nozawa,3 Koichi Fujii,3 Toshihiko Taguchi6 1Public Health and Epidemiology, Meiji Pharmaceutical University, Tokyo, Japan; 2Health Economics and ...
Akazawa M +8 more
doaj
Can Quality-Adjusted Life Year Avoidance Help in Oncology Drug Reimbursement Decisions? [PDF]
John E. Calfee
openalex +1 more source
EE291 Ethical Implications of Quality-Adjusted Life Year Assessments for Patients with Disabilities: A Duchenne Muscular Dystrophy Case Study [PDF]
Alexa C. Klimchak +3 more
openalex +1 more source
SNUPN‐Related Muscular Dystrophy: Novel Phenotypic, Pathological and Functional Protein Insights
ABSTRACT Objective SNUPN‐related muscular dystrophy or LGMDR29 is a new entity that covers from a congenital or childhood onset pure muscular dystrophy to more complex phenotypes combining neurodevelopmental features, cataracts, or spinocerebellar ataxia. So far, 12 different variants have been described.
Nuria Muelas +18 more
wiley +1 more source
ABSTRACT Introduction Neuronal pentraxin 2 (NPTX2) is a synaptic protein involved in synaptic plasticity and regulation of neuronal excitability. Lower baseline cerebrospinal fluid (CSF) NPTX2 levels have been shown to be associated with an earlier onset of mild cognitive impairment (MCI), a pre‐dementia syndrome, even after CSF Alzheimer's Disease (AD)
Juan P. Vazquez +12 more
wiley +1 more source
The effect of comorbidity on health-related quality of life for injury patients in the first year following injury: comparison of three comorbidity adjustment approaches [PDF]
Juanita A. Haagsma +5 more
openalex +1 more source
ABSTRACT Background Neurodegeneration with brain iron accumulation (NBIA) comprises a genetically and clinically heterogeneous group of rare neurological disorders characterized particularly by iron accumulation in the basal ganglia. To date, 15 genes have been associated with NBIA.
Seda Susgun +95 more
wiley +1 more source
Amyotrophic Lateral Sclerosis Prevalence Projection in 2040: A Less Rare Disease
ABSTRACT Objective To project ALS prevalence across multiple countries through 2040, accounting for both population aging and increased survival. Methods Data from the Piemonte and Valle d'Aosta ALS register (PARALS) was used to estimate the trends in incidence and prevalence from 2005 to 2019. Survival trends over this period were also assessed.
Rosario Vasta +18 more
wiley +1 more source

