Onasemnogene Abeparvovec in Patients With SMA: Interim Results of the RESTORE Registry in Japan
ABSTRACT Objective There are limited real‐world data regarding the safety and effectiveness of onasemnogene abeparvovec (OA; Zolgensma) infusion, a one‐time gene replacement therapy, for Japanese patients with spinal muscular atrophy (SMA). We aimed to improve understanding of the real‐world outcomes for OA in Japan.
Kayoko Saito +8 more
wiley +1 more source
Physicochemical Characterization and Metabolites Identification of the Synthetic Cannabinoid MDMB-5'Br-PINACA Using <i>In Silico</i> and <i>In Vitro</i> Approaches. [PDF]
Godoi AB +5 more
europepmc +1 more source
Cognitive and Neuroimaging Divergence Between Juvenile and Adult FUS Amyotrophic Lateral Sclerosis
ABSTRACT Objective Amyotrophic lateral sclerosis (ALS) is a neurodegenerative disorder characterized by progressive motor neuron degeneration. Fused in sarcoma (FUS)‐associated juvenile ALS (jALS) represents a distinct and aggressive subgroup with rapid deterioration and poor prognosis.
Alexandra V. Jürs +7 more
wiley +1 more source
A new species of Mariauxiella de Chambrier & Rego, 1995 (Cestoda: Proteocephalidae) infecting the driftwood catfish Ageneiosus militaris Valenciennes (Siluriformes: Auchenipteridae) in Brazil. [PDF]
Alves PV, Rocha HL, da Silva RJ.
europepmc +1 more source
Characterizing Cutaneous α‐Synuclein Deposition and Seeding Activity in Parkinson's Disease Subtypes
ABSTRACT Objective Cutaneous phosphorylated α‐synuclein (p‐syn) and α‐synuclein seeding activity are promising biomarkers for Parkinson's disease (PD), but their clinical value remains uncertain due to disease heterogeneity. This study evaluates these two biomarkers in PD patients to inform phenotype‐specific diagnosis and disease severity assessment ...
Yuting Jin +8 more
wiley +1 more source
[Paradigm shift in modern palliative care: the need for a multidimensional approach]. [PDF]
Prats Orvay M, Lozano A.
europepmc +1 more source
Natural Frequencies of Levodopa‐Induced Dyskinesia in Parkinson's Disease
ABSTRACT Objectives Abnormal involuntary movements, known as dyskinesias, are common complications of levodopa treatment in patients with Parkinson's disease and can significantly impair quality of life. The underlying pathophysiology remains unclear, and current therapeutic options are limited.
Ioannis U. Isaias +3 more
wiley +1 more source
Modulating Luminescence Thermometry of the Eu<sup>III</sup>/Tb<sup>III</sup> Pair in Coordination Polymers through Ligand Structure and Lanthanide(III) Molar Ratio. [PDF]
Araujo GJS +10 more
europepmc +1 more source
ABSTRACT Background Hereditary Spastic Paraplegia (HSP) comprises a group of rare genetic diseases characterized by length‐dependent axonal degeneration of the corticospinal tracts and dorsal columns, whose main clinical feature is spastic gait. Pathogenic variants in the SPG4 gene cause Spastic Paraplegia Type 4 (SPG4‐HSP), the most common form of HSP.
Gaia Fattorini +12 more
wiley +1 more source

