Patient‐driven search for rare disease therapies: the Fondazione Telethon success story and the strategy leading to Strimvelis
Author:
Affiliation:
1. Fondazione Telethon Milan Italy
Publisher
EMBO
Subject
Molecular Medicine
Link
https://onlinelibrary.wiley.com/doi/pdf/10.15252/emmm.201607293
Reference8 articles.
1. Gene Therapy for Immunodeficiency Due to Adenosine Deaminase Deficiency
2. Fighting Rare Diseases: The Model of the Telethon Research Institutes in Italy
3. LMO2 -Associated Clonal T Cell Proliferation in Two Patients after Gene Therapy for SCID-X1
4. Reviewing in science requires quality criteria and professional reviewers
5. The quiet rise of the NIH’s hot new metric
Cited by 16 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. Preventive pharmacological treatment in subjects at risk for fatal familial insomnia: science and public engagement;Prion;2022-06-23
2. Fondazione Telethon and Unione Italiana Lotta alla Distrofia Muscolare, a successful partnership for neuromuscular healthcare research of value for patients;Orphanet Journal of Rare Diseases;2021-10-02
3. Plant Viruses and Bacteriophage-Based Reagents for Diagnosis and Therapy;Annual Review of Virology;2020-09-29
4. An “all-wheel drive” proposal to accelerate clinical research in common and rare neurological diseases;Neurological Sciences;2019-12-19
5. EU Regulatory Pathways for ATMPs: Standard, Accelerated and Adaptive Pathways to Marketing Authorisation;Molecular Therapy - Methods & Clinical Development;2019-06
1.学者识别学者识别
2.学术分析学术分析
3.人才评估人才评估
"同舟云学术"是以全球学者为主线,采集、加工和组织学术论文而形成的新型学术文献查询和分析系统,可以对全球学者进行文献检索和人才价值评估。用户可以通过关注某些学科领域的顶尖人物而持续追踪该领域的学科进展和研究前沿。经过近期的数据扩容,当前同舟云学术共收录了国内外主流学术期刊6万余种,收集的期刊论文及会议论文总量共计约1.5亿篇,并以每天添加12000余篇中外论文的速度递增。我们也可以为用户提供个性化、定制化的学者数据。欢迎来电咨询!咨询电话:010-8811{复制后删除}0370
www.globalauthorid.com
TOP
Copyright © 2019-2024 北京同舟云网络信息技术有限公司 京公网安备11010802033243号 京ICP备18003416号-3