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.

Comment

Annemieke Aartsma-Rus et al. Lancet Neurol. 2017 Apr.

. 2017 Apr;16(4):264.

doi: 10.1016/S1474-4422(17)30041-8.

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.

Erratum in

Corrections.
[No authors listed] [No authors listed] Lancet Neurol. 2017 May;16(5):341. doi: 10.1016/S1474-4422(17)30090-X. Lancet Neurol. 2017. PMID: 28414644 No abstract available.

No abstract available

Stakeholder cooperation to overcome challenges in orphan medicine development: the example of Duchenne muscular dystrophy.
Straub V, Balabanov P, Bushby K, Ensini M, Goemans N, De Luca A, Pereda A, Hemmings R, Campion G, Kaye E, Arechavala-Gomeza V, Goyenvalle A, Niks E, Veldhuizen O, Furlong P, Stoyanova-Beninska V, Wood MJ, Johnson A, Mercuri E, Muntoni F, Sepodes B, Haas M, Vroom E, Aartsma-Rus A. Straub V, et al. Lancet Neurol. 2016 Jul;15(8):882-890. doi: 10.1016/S1474-4422(16)30035-7. Lancet Neurol. 2016. PMID: 27302365 Review.