Addressing the challenges of novel therapies in rare diseases with mechanistic perspectives: Missed opportunities or the way forward?

Br J Clin Pharmacol. 2022 Jun;88(6):2480-2483. doi: 10.1111/bcp.15350. Epub 2022 Apr 21.

Authors

Janet Mifsud¹, Noel Cranswick²

Affiliations

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.

PMID: 35446442
DOI: 10.1111/bcp.15350

No abstract available

Publication types

Editorial

MeSH terms

Humans
Rare Diseases* / drug therapy