Risdiplam treatment has not led to retinal toxicity in patients with spinal muscular atrophy-Reference-Cited by-同舟云学术

Risdiplam treatment has not led to retinal toxicity in patients with spinal muscular atrophy

Published:2020-11-24 Issue:1 Volume:8 Page:54-65
ISSN:2328-9503
Container-title:Annals of Clinical and Translational Neurology
language:en
Short-container-title:Ann. Clin. Transl. Neurol.

Author:

Sergott Robert C.¹²,Amorelli Giulia M.³,Baranello Giovanni⁴⁵,Barreau Emmanuel⁶,Beres Shannon⁷,Kane Steven⁸,Mercuri Eugenio³^ORCID,Orazi Lorenzo³,SantaMaria Melissa²,Tremolada Gemma⁹,Santarsiero Diletta⁹,Waskowska Agnieszka¹⁰,Yashiro Shigeko¹¹,Denk Nora¹²,Fürst‐Recktenwald Sabine¹³,Gerber Marianne¹⁴^ORCID,Gorni Ksenija¹⁵,Jaber Birgit¹⁴,Jacobsen Bjoern¹²,Mueller Lutz¹²,Nave Stephane¹²,Scalco Renata S.¹³,Marzoli Stefania B.⁹,

Affiliation:

1. Department of Neuro‐Ophthalmology Wills Eye Hospital Philadelphia USA

2. Annesley EyeBrain Center Thomas Jefferson University Philadelphia USA

3. Paediatric Neurology and Nemo Center Catholic University and Policlinico Gemelli Rome Italy

4. The Dubowitz Neuromuscular Centre NIHR Great Ormond Street Hospital Biomedical Research CentreGreat Ormond Street Institute of Child Health University College London& Great Ormond Street Hospital Trust London UK

5. Developmental Neurology Unit Fondazione IRCCS Istituto Neurologico Carlo Besta Milan Italy

6. Institute of Myology Paris France

7. Department of Neurology Department of Ophthalmology Stanford University Palo Alto California USA

8. Columbia University Medical Center New York USA

9. Neuro‐Ophthalmology Center Ophthalmology Department IRCCS Istituto Auxologico Italiano Milan Italy

10. Department of Developmental Neurology Medical University of Gdańsk Gdańsk Poland

11. Department of Ophthalmology National Center for Global Health and Medicine (NCGM) Tokyo Japan

12. Roche Pharma Research and Early Development Pharmaceutical Sciences Roche Innovation Center Basel, F. Hoffmann‐La Roche Ltd Basel Switzerland

13. Pharma Development Neurology F. Hoffmann‐La Roche Ltd Basel Switzerland

14. Pharma DevelopmentSafetyF. Hoffmann‐La Roche Ltd Basel Switzerland

15. PDMA Neuroscience and Rare DiseaseF. Hoffmann‐La Roche Ltd Basel Switzerland

Funder

F. Hoffmann-La Roche

Publisher

Wiley

Subject

Clinical Neurology,General Neuroscience

Link

https://onlinelibrary.wiley.com/doi/pdf/10.1002/acn3.51239

Reference22 articles.

1. The Genetics of Spinal Muscular Atrophy: Progress and Challenges

2. Childhood spinal muscular atrophy: controversies and challenges

3. The role of survival motor neuron protein (SMN) in protein homeostasis

4. A single nucleotide in the SMN gene regulates splicing and is responsible for spinal muscular atrophy

5. Prevalence, incidence and carrier frequency of 5q–linked spinal muscular atrophy – a literature review

Cited by 28 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. Exploration of adverse events associated with risdiplam use: Retrospective cases from the US Food and Drug Administration Adverse Event Reporting System (FAERS) database;PLOS ONE;2024-03-01

2. Consensus from the Brazilian Academy of Neurology for the diagnosis, genetic counseling, and use of disease-modifying therapies in 5q spinal muscular atrophy;Arquivos de Neuro-Psiquiatria;2024-01

3. The Eye;Haschek and Rousseaux' s Handbook of Toxicologic Pathology;2024

4. Risdiplam for the treatment of spinal muscular atrophy;Zhurnal nevrologii i psikhiatrii im. S.S. Korsakova;2024

5. SMALL-MOLECULE RNA-SPLICING MODULATORS;Medicinal Chemistry Reviews;2023-11-16