Implementing a Global Expanded Access Program (EAP) for Infantile-Onset Spinal Muscular Atrophy (Type I): Understanding the Imperative, Impact and Challenges-Reference-Cited by-同舟云学术

Implementing a Global Expanded Access Program (EAP) for Infantile-Onset Spinal Muscular Atrophy (Type I): Understanding the Imperative, Impact and Challenges

Published:2019-05-21 Issue:2 Volume:6 Page:227-231
ISSN:2214-3599
Container-title:Journal of Neuromuscular Diseases
language:
Short-container-title:JND

Author:

Yong Jonathan¹,Moffett Megan¹,Lucas Sam¹

Affiliation:

1. Biogen, Cambridge, MA, USA

Publisher

IOS Press

Subject

Clinical Neurology,Neurology

Reference20 articles.

1. Newborn and carrier screening for spinal muscular atrophy;Prior;Am J Med Genet A,2010

2. Spinal muscular atrophy;Lunn;Lancet,2008

3. A population-based study of genotypic and phenotypic variability in children with spinal muscular atrophy;Arkblad;Acta Paediatr,2009

4. Incidence of spinal muscular atrophy in Poland–more frequent than predicted?;Jedrzejowska;Neuroepidemiology,2010

5. 209th ENMC International Workshop: Outcome Measures and Clinical Trial Readiness in Spinal Muscular Atrophy 7–9 November 2014, Heemskerk, The Netherlands;Finkel;Neuromuscul Disord,2015

Cited by 9 articles. 订阅此论文施引文献订阅此论文施引文献，注册后可以免费订阅5篇论文的施引文献，订阅后可以查看论文全部施引文献

1. Global Risdiplam Compassionate Use Program for Patients with Type 1 or 2 Spinal Muscular Atrophy;Clinical Therapeutics;2024-04

2. Literature Review on Health Emigration in Rare Diseases—A Machine Learning Perspective;International Journal of Environmental Research and Public Health;2023-01-30

3. Adherence and Persistence to Nusinersen for Spinal Muscular Atrophy: A US Claims-Based Analysis;Advances in Therapy;2022-12-19

4. Canadian healthcare capacity gaps for disease-modifying treatment in Huntington’s disease: a survey of current practice and modelling of future needs;BMJ Open;2022-06

5. An expanded access program of risdiplam for patients with Type 1 or 2 spinal muscular atrophy;Annals of Clinical and Translational Neurology;2022-05-14