This Review highlights emerging RNA-targeted therapeutics for gene activation, focusing on opportunities and challenges for translation to the clinic.
Monday, June 5, 2023
Amplifying gene expression with RNA-targeted therapeutics
Khorkova O, Stahl J, Joji A, Volmar CH, Wahlestedt C. Amplifying gene expression with RNA-targeted therapeutics. Nature reviews. Drug Discovery. 2023 May. DOI: 10.1038/s41573-023-00704-7. PMID: 37253858; PMCID: PMC10227815.
Anaesthetic management of thyroid storm in a patient with Friederich’s ataxia. A case report
M. Sneyers Closa, A. Pérez Requena, S. Sánchez García, J. Sistac Ballarín, Anaesthetic management of thyroid storm in a patient with Friederich’s ataxia. A case report, Revista Española de Anestesiología y Reanimación (English Edition), 2023, doi:10.1016/j.redare.2023.06.002.
A 26-year-old patient with Friederich's ataxia with hypertrophic obstructive cardiomyopathy undergoing total thyroidectomy due to persistent amiodarone-induced thyrotoxicosis (despite high doses of antithyroid drugs and corticosteroids), presented an intraoperative episode suggestive of thyroid storm. Thyroid storm is an endocrine emergency that is associated with high morbidity and mortality. Early diagnosis and treatment, which is of vital importance to improve survival, includes symptomatic treatment, treatment of cardiovascular, neurological, and/or hepatic manifestations and thyrotoxicosis, measures to suppress or avoid triggering stimuli, and definitive treatment.
Paciente de 26 años afecto de ataxia de Friederich con una miocardiopatía hipertrófica obstructiva sometido a una tiroidectomía total por tirotoxicosis secundaria a amiodarona persistente (a pesar de elevadas dosis de antitiroideos y corticoides) que intraoperatoriamente presentó un episodio sugestivo de tormenta tiroidea. La tormenta tiroidea es una emergencia endocrinológica que asocia una elevada morbimortalidad. Para mejorar la supervivencia es de vital importancia un diagnóstico y tratamiento precoz que incluya: un tratamiento sintomático; el tratamiento de las manifestaciones cardiovasculares, neurológicas y/o hepáticas y de la tirotoxicosis; y así como suprimir o evitar estímulos desencadenantes y practicar un tratamiento definitivo.
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