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Title: | Addressing the challenges of novel therapies in rare diseases with mechanistic perspectives : missed opportunities or the way forward? |
Authors: | Mifsud, Janet Cranswick, Noel |
Keywords: | Editorials Drugs -- Toxicology Rare diseases -- Treatment Rare diseases -- Diagnosis Rare diseases -- Patients |
Issue Date: | 2022 |
Publisher: | British Pharmacological Society |
Citation: | Mifsud, J., & Cranswick, N. (2022). Addressing the challenges of novel therapies in rare diseases with mechanistic perspectives: Missed opportunities or the way forward?. British Journal of Clinical Pharmacology, 1-4. 10.1111/bcp.15350 |
Abstract: | There is no universally agreed definition of what is a rare disease. Depending upon the jurisdiction, the incidence of rare is defined as occurring in less than one in 1500 (in the United States) to one in 2000 (European Union). Rare Diseases Day falls on the 29 February but often celebrated on 28 February during a non-leap year. While individually such conditions are clearly uncommon, with approximately 8000 rare diseases identified, they cumulatively encompass a significant portion of the population. It is estimated that, worldwide, there are 400 million people living with a rare disease. Rare diseases have, in the past, been largely neglected by the pharmaceutical industry who calculated that the development of a therapy for an uncommon indication was unlikely to be profitable. This lacuna was recognised by governments internationally with the enactment of the Orphan Drug Act in the United States in 1983, and the EU orphan drug designation 2000. This led to huge impetus towards the discovery of new therapies for rare diseases.1 This special issue of the British Journal of Clinical Pharmacology Reviews, in fact, attempts to address the knowledge gained in the last few decades on novel perspectives and mechanism in the search for new potential treatments for rare diseases. The issue includes six papers encompassing a variety of rare diseases where the authors have used varying perspectives in the identification of innovative tools in this quest. |
URI: | https://www.um.edu.mt/library/oar/handle/123456789/94821 |
Appears in Collections: | Scholarly Works - FacM&SCPT |
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File | Description | Size | Format | |
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Addressing the challenges of novel therapies in rare diseases with mechanistic perspectives.pdf Restricted Access | 327.08 kB | Adobe PDF | View/Open Request a copy |
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