Results 241 to 250 of about 48,155 (306)

Therapeutic Effects of Nizubaglustat in a Mouse Model of GM2 Gangliosidosis

open access: yesJournal of Inherited Metabolic Disease, Volume 49, Issue 1, January 2026.
ABSTRACT Nizubaglustat is a novel selective inhibitor of glucosylceramide synthase (GCS) and the non‐lysosomal glucocerebrosidase (NLGase, GbA2) with brain penetrant properties. It is currently in clinical development as an oral treatment for rare lysosomal storage diseases with neurological involvement. One such disease group called GM2 gangliosidosis,
Kyle Landskroner   +3 more
wiley   +1 more source

From Molecule to Meaning: Neuronopathic Biomarkers and Clinical Relevance in GM1

open access: yesJournal of Inherited Metabolic Disease, Volume 49, Issue 1, January 2026.
ABSTRACT GM1 gangliosidosis is a rare, progressively neurodegenerative lysosomal storage disorder characterized by profound central nervous system involvement and substantial clinical heterogeneity. The development of reliable biomarkers is essential for tracking disease progression, stratifying patients, and advancing clinical trial readiness. Primary
Krista Casazza   +3 more
wiley   +1 more source

Lumbar Puncture and Spinal Analgesia

open access: yesBritish Journal of Anaesthesia, 1973
openaire   +1 more source

Ballistics and wound ballistics of CO2‐powered dart guns in relation to ungulate dart injury and animal welfare

open access: yesWildlife Biology, Volume 2026, Issue 1, January 2026.
Large mammals are darted with dart guns to inject drugs or to collect biopsy tissue for capture, research, or veterinary purposes, especially for procedures involving wild and zoo animals. Darting is invasive and associated with risks of trauma, and severe and fatal dart injuries have been reported.
Jörg Beckmann   +2 more
wiley   +1 more source

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