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Methodological challenges in outcomes research for early-trials for implementation of new therapies in neuropediatric rare diseases.

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Title: Methodological challenges in outcomes research for early-trials for implementation of new therapies in neuropediatric rare diseases.
Authors: Acosta, Maria T.1 (AUTHOR) acostam@nhgri.nih.gov, Zaragoza Domingo, Silvia2 (AUTHOR) szaragoza@psyncro.net, Arango, Celso3 (AUTHOR) carango@hggm.es, Bishop, Kim I.4 (AUTHOR) kib@globalpharmaconsultancy.com, Busner, Joan5 (AUTHOR) joan.busner@signanthealth.com, Dorffner, Georg6 (AUTHOR) georg.dorffner@thesiestagroup.com, Cerro, Inés del7 (AUTHOR) ines.dcsi@gmail.com, Diaz, John Carlos8 (AUTHOR) johndiaz@geosera.com, Gruenblatt, Edna9 (AUTHOR) edna.gruenblatt@kjpd.uzh.ch, Hölter, Sabine M.10 (AUTHOR) sabine.hoelter-koch@helmholtz-munich.de, Harel, Brian11 (AUTHOR) brian.harel@takeda.com, Krishna, Vanitha11 (AUTHOR) vanitha.krishna@gmail.com, Mas, Marta12 (AUTHOR) davitito86@gmail.com, Moreno, Carmen3 (AUTHOR) cmoreno@hggm.es, Pallanti, Stefano13 (AUTHOR) s.pallanti@istitutodineuroscienze.it, Plasencia, Carme14 (AUTHOR) carmen.plasencia@aromics.es, Kittel-Schneider, Sarah15 (AUTHOR) SKittelSchneider@ucc.ie, Tinoco, Daniella16 (AUTHOR) dtinoco@hotmail.com, Vance, Monica17 (AUTHOR) mvance@santium.com, Singh, Manpreet K.18 (AUTHOR) mpksingh@ucdavis.edu
Source: Orphanet Journal of Rare Diseases. 10/17/2025, Vol. 20 Issue 1, p1-7. 7p.
Database: Academic Search Ultimate
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ISSN:17501172
DOI:10.1186/s13023-025-03814-0