Stakeholder collaboration for spinal muscular atrophy therapy development

Affiliations

• ¹ Leiden University Medical Center, Leiden, Netherlands. Electronic address: a.m.rus@lumc.nl.
• ² European Medicines Agency, Human Medicines Evaluation Division, Scientific and Regulatory Department, Central Nervous System and Ophthalmology Office, London, UK.
• ³ Famiglie Sma, Milan, Italy.
• ⁴ Bundesinstitut für Arzneimittel und Medizinprodukte, 53175 Bonn, Germany.
• ⁵ The SMA Trust, Stratford upon Avon, UK.
• ⁶ Laboratório de Farmacologia Clínica e Terapêutica, Faculdade de Medicina, Universidade de Lisboa, Lisboa, Portugal.
• ⁷ The Dubowitz Neuromuscular Centre, University College London Great Ormond Street Institute of Child Health and Great Ormond Street Hospital, London, UK.
• ⁸ Nemours Children's Hospital and University of Central Florida College of Medicine, Orlando, USA.
• ⁹ Catholic University Rome, Italy.