Pat Furlong
Parent Project Muscular Dystrophy(US)
Publications by Year
Research Areas
Muscle Physiology and Disorders, Neurogenetic and Muscular Disorders Research, Biomedical Ethics and Regulation, Genetic Neurodegenerative Diseases, Biomedical and Engineering Education
Most-Cited Works
- → Large-scale serum protein biomarker discovery in Duchenne muscular dystrophy(2015)291 cited
- → Stakeholder cooperation to overcome challenges in orphan medicine development: the example of Duchenne muscular dystrophy(2016)92 cited
- → Repeated intravenous cardiosphere-derived cell therapy in late-stage Duchenne muscular dystrophy (HOPE-2): a multicentre, randomised, double-blind, placebo-controlled, phase 2 trial(2022)80 cited
- → A phase 3 randomized placebo-controlled trial of tadalafil for Duchenne muscular dystrophy(2017)73 cited
- → Recommendations for the development of rare disease drugs using the accelerated approval pathway and for qualifying biomarkers as primary endpoints(2015)57 cited
- → Measuring clinical effectiveness of medicinal products for the treatment of Duchenne muscular dystrophy(2014)45 cited
- → How a patient advocacy group developed the first proposed draft guidance document for industry for submission to the U.S. Food and Drug Administration(2015)44 cited
- → Mothers’ psychological adaptation to Duchenne/Becker muscular dystrophy(2015)43 cited
- → Report on the workshop: Meaningful outcome measures for Duchenne muscular dystrophy, London, UK, 30–31 January 2017(2018)36 cited
- → Delays in diagnosis of Duchenne muscular dystrophy: An evaluation of genotypic and sociodemographic factors(2019)27 cited