Latest Articles
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Randomized Controlled Trial Multicenter Study
Effects of eplontersen on symptoms of autonomic neuropathy in hereditary transthyretin-mediated amyloidosis: secondary analysis from the NEURO-TTRansform trial.
The NEURO-TTRansform trial showed that after 66 weeks of treatment, eplontersen significantly reduced neuropathic impairment and improved quality of life (QoL) in patients with hereditary transthyretin-mediated amyloidosis with polyneuropathy (ATTRv-PN). In this secondary analysis from NEURO-TTRansform, autonomic impairment, and the impact of eplontersen on autonomic impairment progression was evaluated through 85 weeks in patients randomised to eplontersen (n = 144) versus external placebo (n = 60; through Week 66 from the NEURO-TTR trial). ⋯ Eplontersen demonstrated benefit across multiple measures of autonomic impairment known to progress rapidly and negatively impact QoL without treatment, without deterioration in nutritional status.
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Med. Clin. North Am. · Mar 2025
ReviewEvaluation and Management of Acute Transient Loss of Consciousness.
Transient loss of consciousness (TLOC) is among the most common complaints encountered by clinicians across specialties. Possible etiologies of acute TLOC can be classified as neurologic, systemic, and functional. ⋯ Functional neurologic disorders (also known as conversion disorders or psychogenic spells) refer to clinical conditions in which clinical presentation can mimic but does not completely fit with any specific neurologic or systemic disorder and conventional diagnostic testing reveals normal neurologic and systemic structures. The focus of this review will be on the diagnostic process and tools to differentiate between the multitudes of causes of TLOC.
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Multicenter Study
PRX004 in variant amyloid transthyretin (ATTRv) amyloidosis: results of a phase 1, open-label, dose-escalation study.
The investigational monoclonal antibody PRX004 is designed to specifically target and deplete TTR amyloid. Here, we report on the safety, tolerability, pharmacokinetics, pharmacodynamics and preliminary clinical activity of PRX004 in patients with ATTRv amyloidosis. ⋯ PRX004 was well tolerated in patients with ATTRv amyloidosis and demonstrated potential clinical activity. A phase 2 randomised controlled trial in ATTR cardiomyopathy is ongoing (NCT05442047).
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Vocal cord disorders present with a variety of symptoms including dysphonia, respiratory symptoms, and stridor. When evaluating symptoms, a complete history and through head, neck, and neurologic examinations are necessary. ⋯ Most masses of the vocal folds are benign and resolve with voice hygiene and speech therapy. Surgery is reserved for persistent symptomatic nodules and cancerous lesions.
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About 85% of US adults with hearing loss have unmet hearing needs, creating significant individual and population effects on chronic conditions, socioeconomics, and quality of life. This article reviews the diagnosis and management of hearing loss, overcoming systemic barriers, resources in limited primary care settings, and a multidisciplinary approach.