Future Rare Diseases

Game Changers for Rare Diseases: A Spotlight on Precision Medicine

We are pleased to announce a new Article Collection in Future Rare Diseases, dedicated to the game-changing role of precision medicine in the management of rare diseases. Precision medicines are revolutionizing patient care, offering significant benefits in outcomes and quality of life for eligible patients. Given the importance of precision medicine in this setting, Future Rare Diseases invites submissions of original research articles, plain language summaries, reviews, and perspectives exploring the mechanisms of action, clinical efficacy, safety, and real-world application of these transformative therapies.

While the call is open to receive manuscripts across the broad spectrum of precision medicine and rare diseases, the Editors are particularly interested in manuscripts relating to the following areas:

• CFTR modulators for cystic fibrosis
• Enzyme replacement & oral chaperone therapies for Fabry’s Disease
• Gene therapies for neuromuscular and neurological conditions including spinal muscular atrophy, Duchenne muscular dystrophy and cerebral adrenoleukodystrophy.
• Gene therapies for haematological conditions including haemophilia A, beta thalassemia and sickle cell disease.
• Drug repurposing as potential treatment for rare diseases

The collection is part of the new Game Changer series of Article Collections, focusing on breakthrough therapies, drugs, or technologies that have significantly altered the standard of care, leading to game-changing improvements in patient outcomes. Papers published within the Game Changer series will benefit from additional promotional activities across Taylor and Francis, increasing the discoverability and visibility of your research.

Submitting authors are eligible for a discount on the Article Publishing charge by applying a discount code at the point of submission. If you have any queries regarding the Article Collection or would like to discuss a contribution, then please email Alex Dearman, Global Head of Content Acquisition, at alexander.dearman@informa.com and they will be happy to assist.