Addressing the challenges of novel therapies in rare diseases with mechanistic perspectives: Missed opportunities or the way forward?
Author:
Affiliation:
1. Department of Clinical Pharmacology and Therapeutics, Faculty of Medicine and Surgery University of Malta Msida Malta
2. Clinical Pharmacology Royal Children's Hospital Melbourne Australia
Publisher
Wiley
Subject
Pharmacology (medical),Pharmacology
Link
https://onlinelibrary.wiley.com/doi/pdf/10.1111/bcp.15350
Reference24 articles.
1. Orphan drug designation in Europe: A booster for the research and development of drugs in rare diseases
2. Incentives for Drug Development — The Curious Case of Colchicine
3. Therapeutic RNA interference: A novel approach to the treatment of primary hyperoxaluria
4. Conventional and novel therapeutic options in children with familial Mediterranean fever: A rare autoinflammatory disease
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