Stakeholder collaboration for spinal muscular atrophy therapy development.
Leiden University Medical Center, Leiden, Netherlands. Electronic address: a.m.rus@lumc.nl.
Annemieke
A
Aartsma-Rus
European Medicines Agency, Human Medicines Evaluation Division, Scientific and Regulatory Department, Central Nervous System and Ophthalmology Office, London, UK.
Pavel
P
Balabanov
Famiglie Sma, Milan, Italy.
Luca
L
Binetti
European Medicines Agency, Human Medicines Evaluation Division, Scientific and Regulatory Department, Central Nervous System and Ophthalmology Office, London, UK.
Manuel
M
Haas
Bundesinstitut für Arzneimittel und Medizinprodukte, 53175 Bonn, Germany.
Marion
M
Haberkamp
The SMA Trust, Stratford upon Avon, UK.
Joanna
J
Mitchell
Laboratório de Farmacologia Clínica e Terapêutica, Faculdade de Medicina, Universidade de Lisboa, Lisboa, Portugal.
Mário Miguel
MM
Rosa
The Dubowitz Neuromuscular Centre, University College London Great Ormond Street Institute of Child Health and Great Ormond Street Hospital, London, UK.
Francesco
F
Muntoni
Nemours Children's Hospital and University of Central Florida College of Medicine, Orlando, USA.
Richard
R
Finkel
Catholic University Rome, Italy.
Eugenio
E
Mercuri
D006801:Humans; D009134:Muscular Atrophy, Spinal; D014897:Spinal Muscular Atrophies of Childhood