Dr Marianela Schiava Robert Muni Lofra Dr John Bourke Meredith James Professor Jordi Diaz Manera et al. | Disease-associated comorbidities, medication records and anthropometric measures in adults with Duchenne muscular dystrophy | 2024 |
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Dr Marianela Schiava Dr John Bourke Meredith James Dr Maha Elseed Dr Monika Malinova et al. | Functional abilities, respiratory and cardiac function in a large cohort of adults with Duchenne muscular dystrophy treated with glucocorticoids | 2024 |
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Victoria Hedley Becca Leary Dr Anando Sen Anna Irvin Emma Heslop et al. | Performing clinical drug trials in children with a rare disease | 2024 |
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Emma Heslop Catherine Turner Anna Irvin Professor Volker Straub Professor Michela Guglieri et al. | Gene therapy in Duchenne muscular dystrophy: Identifying and preparing for the challenges ahead | 2021 |
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Ben Porter Phillip Cammish Joseph Orrell Emma Heslop Professor Chiara Marini Bettolo et al. | The UK FSHD Patient Registry: A Key Tool in the Facilitation of Clinical Research | 2019 |
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Ben Porter Phillip Cammish Emma Heslop Professor Chiara Marini Bettolo
| The UK Myotonic Dystrophy Patient Registry: A Key Tool in the Facilitation of Clinical Research | 2019 |
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Emma Heslop Professor Volker Straub Emerita Professor Katherine Bushby
| The TREAT-NMD advisory committee for therapeutics (TACT): an innovative de-risking model to foster orphan drug development | 2015 |
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Emma Heslop Professor Volker Straub Dr Louise Johnston Emerita Professor Katherine Bushby
| The TREAT-NMD Advisory Committee for Therapeutics (TACT): An innovative de-risking model to foster orphan drug development | 2015 |
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Rachel Thompson Dr Louise Johnston Dr Monica Ensini Emma Heslop Emerita Professor Katherine Bushby et al. | RD-Connect: An Integrated Platform Connecting Databases, Registries, Biobanks and Clinical Bioinformatics for Rare Disease Research | 2014 |
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Dr Pauline McCormack Emeritus Professor Simon Woods Emma Heslop Professor Volker Straub Emerita Professor Katherine Bushby et al. | Guidance in social and ethical issues related to clinical, diagnostic care and novel therapies for hereditary neuromuscular rare diseases: "translating" the translational | 2013 |
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Emma Heslop Dr Pauline McCormack Professor Christoph Rehmann-Sutter Emeritus Professor Simon Woods
| The risks of therapeutic misconception and individual patient (n = 1) “trials” in rare diseases such as Duchenne dystrophy | 2011 |
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Emerita Professor Katherine Bushby Emma Heslop Dr Pauline McCormack Emeritus Professor Simon Woods
| The development of antisense oligonucleotide therapies for Duchenne muscular dystrophy: Report on a TREAT-NMD workshop hosted by the European Medicines Agency (EMA), on September 25th 2009 | 2010 |
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