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CPX-351: From preclinical studies to future directions
作者A. Sperotto; G. Ciotti; M. Basso; M. Gottardi
作者单位UOSD Terapie Cellulari – Dipartimento di Oncologia, Istituto Oncologico Veneto (IOV) – IRCCS – Padova, Italy; UOC Oncoematolgia – Dipartimento di Oncologia, Istituto Oncologico Veneto (IOV) – IRCCS – Padova, Italy; UOC Farmacia – Dipartimento di Oncologia traslazionale e dei servizi trasversali, Istituto Oncologico Veneto (IOV) – IRCCS – Padova, Italy
Real-world evidence supporting orphan drugs approvals for rare neuromuscular disorders in the European Union and the United States: Review of public assessment reports (2015–2025)
作者Beliu García-Parra; Josep M. Guiu; Mónica Povedano; Pilar Modamio
作者单位Doctoral Program in Drug Research, Development and Control, Faculty of Pharmacy and Food Sciences, University of Barcelona, Barcelona, Spain; Clinical Neurophysiology Section – Neurology Service, Bellvitge University Hospital - Bellvitge Health Sciences Campus, University of Barcelona, L'Hospitalet de Llobregat, Barcelona, Spain; Clinical Pharmacy and Pharmaceutical Care Unit, Department of Pharmacy and Pharmaceutical Technology, and Physical Chemistry, Faculty of Pharmacy and Food Sciences, University of Barcelona, Barcelona, Spain; Consortium of Health and Social Care of Catalonia, Barcelona, Spain; Motor Neuron Diseases Unit, Bellvitge University Hospital - Bellvitge Health Sciences Campus, University of Barcelona, L'Hospitalet de Llobregat, Barcelona, Spain
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