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
An AI-driven conceptual framework for detecting fake news and deepfake content: a systematic review. [PDF]
Moyo BV +3 more
europepmc +1 more source
Prominent Movement Disorders in RNU2‐2‐Related Spliceosomopathy
ABSTRACT Pediatric movement disorders often overlap with neurodevelopmental diseases, suggesting shared molecular mechanisms. Variants in small nuclear RNA (snRNA) genes encoding spliceosome components have recently been associated with neurodevelopmental disorders, termed “RNUopathies.” We analyzed genome sequencing data from 14 patients with ...
Magdalena Krygier +6 more
wiley +1 more source
Safety evaluation of the food enzyme arabinan endo-1,5-α-l-arabinanase from the non-genetically modified <i>Aspergillus tubingensis</i> strain GPA41. [PDF]
EFSA Panel on Food Enzymes (FEZ) +17 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
Safety evaluation of an extension of use of the food enzyme glucan 1,4-α-maltohydrolase from the genetically modified <i>Bacillus licheniformis</i> strain NZYM-CY. [PDF]
EFSA Panel on Food Enzymes (FEZ) +15 more
europepmc +1 more source
ABSTRACT Paramagnetic rim lesions (PRLs) and choroid plexus (CP) enlargement reflect smoldering inflammation in multiple sclerosis. Their role in cognitive progression remains unexplored. Eighty‐seven early relapsing–remitting MS patients were enrolled at diagnosis and followed longitudinally.
Stefano Ziccardi +15 more
wiley +1 more source
Safety evaluation of an extension of use of the food enzyme glucan 1,4-α-maltohydrolase from the genetically modified <i>Bacillus licheniformis</i> strain NZYM-FR. [PDF]
EFSA Panel on Food Enzymes (FEZ) +15 more
europepmc +1 more source
ABSTRACT Introduction/Objective Acute intracranial stenting during endovascular thrombectomy (EVT) for ischemic stroke requires intraprocedural antiplatelet therapy (APT) to maintain patency. However, the hemorrhagic risk of combining APT with intravenous thrombolysis (IVT) remains uncertain.
Aaron Rodriguez‐Calienes +75 more
wiley +1 more source
Updated safety evaluation of the food enzyme glucan 1,4-α-maltohydrolase from the genetically modified <i>Bacillus subtilis</i> strain BABSC. [PDF]
EFSA Panel on Food Enzymes (FEZ) +15 more
europepmc +1 more source

