| Literature DB >> 31744015 |
Astrid Pechmann1, Matthias Baumann2, Günther Bernert3, Marina Flotats-Bastardas4, Ursula Gruber-Sedlmayr5, Maja von der Hagen6, Oswald Hasselmann7, Elke Hobbiebrunken8, Veronka Horber9, Jessika Johannsen10, Anna Kellersmann3, Cornelia Köhler11, Arpad von Moers12, Wolfgang Müller-Felber13, Barbara Plecko5, Christof Reihle14, Kurt Schlachter15, Gudrun Schreiber16, Oliver Schwartz17, Martin Smitka6, Elisabeth Steiner18, Corinna Stoltenburg19, Burkhard Stüve20, Manuela Theophil12, Claudia Weiß19, Gert Wiegand21, Ekkehard Wilichowski8, Benedikt Winter22, Wolfgang Wittmann23, Ulrike Schara24, Janbernd Kirschner1,25.
Abstract
The natural history of patients with spinal muscular atrophy (SMA) has changed due to advances in standard care and development of targeted treatments. Nusinersen was the first drug approved for the treatment of all SMA patients. The transfer of clinical trial data into a real-life environment is challenging, especially regarding the advice of patients and families to what extent they can expect a benefit from the novel treatment. We report the results of a modified Delphi consensus process among child neurologists from Germany, Austria and Switzerland about the indication or continuation of nusinersen treatment in children with SMA type 1 based on different clinical case scenarios.Entities:
Year: 2020 PMID： 31744015 DOI： 10.3233/JND-190441
Source DB: PubMed Journal: J Neuromuscul Dis