The Medicines and Healthcare products Regulatory Agency (MHRA) has published a new framework signalling a significant overhaul of how rare disease treatments are tested, approved, and delivered in the UK.
Gillian Molloy, Director of Market Access at AscellaHealth, examines the rare disease landscape in the UK, including progress in improving patient care and challenges in diagnostics and the development of new treatments.
In this interview, Abbas Kanani, MRPharmS, Superintendent Pharmacist at Chemist Click, tells us about amyloidosis, a group of rare diseases in which abnormal proteins deposit as amyloid in tissues and organs.
Craig Caceci, Managing Director, Terebellum®, walks us through how to overcome the cost challenge of CGT development for rare, orphan and genetic therapies.
Pascale V Guillot, at the University College London, looks toward improving the quality of life for those with Osteogenesis imperfecta, a chronic health condition called brittle bone disease.
Open Access Government explore the research efforts of the U.S. National Institutes of Health to mitigate the often-underestimated burden of rare diseases on patients and their families.
Dr Michael Wangler, Assistant Professor at Molecular and Human Genetics Baylor College of Medicine and Katie Sacra, Director of Family Programs, Global Foundation for Peroxisomal Disorders, mother of TJ, discusses the impact of the COVID-19 pandemic on families with rare disease - especially peroxisomal disorders.
Dr Peter Diamond, Head of Research from the Leukaemia Foundation, describes what we need to know about amyloidosis, a rare but devastating blood condition
Dr Dan Williams, SynaptixBio CEO, discusses the search to find the first treatment for one of the world’s rarest diseases, TUBB4A-associated leukodystrophy
Afshin Attari, Senior Director of Public Sector & Unified Platforms at Exponential-e, describes driving the digital-first future of pathology through partnerships.
Mark Daly, Director of the Institute for Molecular Medicine Finland (FIMM), HiLIFE at the University of Helsinki, shares his expertise on medical genetic research on a population scale in Finland.
