A Stanford-led phase 3 trial shows gene therapy skin grafts significantly heal chronic wounds in patients with severe epidermolysis bullosa, reducing pain and improving quality of life.
There is a beacon of hope for patients with a rare genetic disorder called Werner Syndrome as researchers unveil a groundbreaking discovery: Nicotinamide riboside, a vitamin B3 derivative, reverses premature ageing.
A new treatment option has become available for severe epilepsy: fenfluramine, which is now available on the NHS for children and adults living with Lennox–Gastaut syndrome (LGS).
Rare Diseases International, whose community engages with the World Health Organization, examines rare diseases and health equity, emphasising shifting the paradigm through global action.
Now is the time to invest in trans-national control strategies to stop the spread of neglected tropical diseases and limit suffering, say Jake Mathewson and Ente Rood, Epidemiologists at KIT Royal Tropical Institute.
February 29th is dedicated to raising awareness about rare diseases and looking into the opportunities technology could bring for potential treatments.
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.
