Longitudinal Metabolomics in Amyotrophic Lateral Sclerosis Implicates Impaired Lipid Metabolism
Objective Amyotrophic lateral sclerosis (ALS) is a fatal neurodegenerative disease characterized by altered metabolome and energy homeostasis, manifesting with body mass index changes and hypermetabolism—both prognostic of disease progression and survival.
Kai Guo+7 more
wiley +1 more source
Post-Marketing Safety of Spinal Muscular Atrophy Therapies: Analysis of Spontaneous Adverse Drug Reactions from EudraVigilance. [PDF]
Belančić A+4 more
europepmc +1 more source
Cyclic Alternating Pattern Dynamics in Individuals at Risk for Developing Parkinson's Disease
Objective The objective of this study was to investigate the differences in cyclic alternating patterns (CAP) metrics, a non‐rapid eye movement (NREM) sleep physiological rhythm, among recently diagnosed patients with Parkinson's disease (PD), and individuals at high and low risk for developing PD based on genetic and prodromal risk.
Andrew Dagay+5 more
wiley +1 more source
Correction: "…They were just treating her Symptom by Symptom": maternal experiences of having a child with spinal muscular atrophy in Ghana. [PDF]
Tawiah ED, Sarfo JO.
europepmc +1 more source
Phosphatemia is an Independent Prognostic Factor in Amyotrophic Lateral Sclerosis
Objective The objective of this study was to evaluate the prognostic value of several muscle damage biomarkers. Methods Data from Piemonte and Valle d'Aosta Amyotrophic Lateral Sclerosis (PARALS) were considered for this study. Survival was defined as the time from diagnosis to death, tracheostomy, or the censoring date.
Rosario Vasta+18 more
wiley +1 more source
Gene therapy for spinal muscular atrophy: timing is key. [PDF]
Servais L.
europepmc +1 more source
Autosomal Recessive Cerebellar Ataxias: Translating Genes to Therapies
Autosomal recessive cerebellar ataxias are disabling neurodegenerative genetic conditions affecting balance and coordination. Advancements in genomic testing have improved diagnosis, leading to a new focus on the development of targeted precision therapeutics addressing cellular, biochemical, and genetic disease mechanisms with a resulting emphasis on ...
Brent L. Fogel+10 more
wiley +1 more source
Onasemnogene-abeparvovec administration to premature infants with spinal muscular atrophy. [PDF]
Brown SM+7 more
europepmc +1 more source
Towards Polymer Composite‐Based Transient Electronic Systems
Biodegradable electronic systems are gaining attention for implantable biomedical applications, targeting disease treatment and lifespan extension. This review covers advances in biocompatible, biodegradable polymer composites using synthetic and natural polymers with conductive, semiconductive, and insulating fillers.
Gwan‐Jin Ko+5 more
wiley +1 more source