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EFAS/EAN survey on the influence of the COVID-19 pandemic on European clinical ...

Fanciulli, Alessandra; Skorić, Magdalena Krbot; Leys, Fabian; Carneiro, Diogo Reis; Campese, Nicole; Calandra-Buonaura, Giovanna; Camaradou, Jennifer

Purpose: To understand the influence of the coronavirus disease 2019 (COVID-19) pandemic on clinical autonomic education and research in Europe. Methods: We invited 84 European autonomic centers to complete an online survey, recorded the pre-pandemic-to-pandemic percentage of junior participants in the annual congresses of the European Federation of Autonomic Societies (EFAS) and European Academy of Neurology (...


Clinical autonomic nervous system laboratories in Europe: a joint survey of the...

Habek, Mario; Leys, Fabian; Krbot Skorić, Magdalena; Reis Carneiro, Diogo; Calandra‐Buonaura, Giovanna; Camaradou, Jennifer; Chiaro, Giacomo

Background and purpose: Disorders of the autonomic nervous system (ANS) are common conditions, but it is unclear whether access to ANS healthcare provision is homogeneous across European countries. The aim of this study was to identify neurology-driven or interdisciplinary clinical ANS laboratories in Europe, describe their characteristics and explore regional differences. Methods: We contacted the European nat...


Long-term efficacy and safety of inotersen for hereditary transthyretin amyloid...

Brannagan, Thomas H.; Coelho, Teresa; Wang, Annabel K.; Polydefkis, Michael J.; Dyck, Peter J.; Berk, John L.; Drachman, Brian; Gorevic, Peter

Background: Hereditary transthyretin amyloidosis (hATTR/ATTRv) results from the deposition of misfolded transthyretin (TTR) throughout the body, including peripheral nerves. Inotersen, an antisense oligonucleotide inhibitor of hepatic TTR production, demonstrated a favorable efficacy and safety profile in patients with the polyneuropathy associated with hATTR in the NEURO-TTR (NCT01737398) study. We report long...


Long-term safety and efficacy of patisiran for hereditary transthyretin-mediate...

Adams, David; Polydefkis, Michael; González-Duarte, Alejandra; Wixner, Jonas; Kristen, Arnt V.; Schmidt, Hartmut H.; Berk, John L.

Background: Hereditary transthyretin-mediated amyloidosis is a rare, inherited, progressive disease caused by mutations in the transthyretin (TTR) gene. We assessed the safety and efficacy of long-term treatment with patisiran, an RNA interference therapeutic that inhibits TTR production, in patients with hereditary transthyretin-mediated amyloidosis with polyneuropathy. Methods: This multicentre, open-label ex...


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