|
Indian Journal of Pharmacology
Medknow Publications on behalf of Indian Pharmacological Society
ISSN: 0253-7613 EISSN: 1998-3751
Vol. 38, Num. 5, 2006, pp. 375-375
|
Indian Journal of Pharmacology, Vol. 38, No. 5, September-October, 2006, pp. 375
Correspondence
Ultra orphan drugs and diseases
Kumar D, Gupta S
Department of Pharmacology and Therapeutics, Govt. Medical College Jammu. (J & K) - 180 001
Correspondence Address:Department of Pharmacology and Therapeutics, Govt. Medical College Jammu. (J & K) - 180 001, drdeepinder@rediffmail.com
Code Number: ph06109
The educational forum, 'Orphan diseases and drugs', (Indian J Pharmacol 2006; 38:171-6) is a very informative article. The various aspects of orphan diseases and drugs have been well covered. A new and interesting term, 'ultra orphan drugs and diseases', was however not included in the article. Ultra orphan drugs[1] is the term designated to drugs which are used to treat exceptionally rare and chronically debilitating or life threatening diseases. Ultra orphan disease is the term designated to diseases with a prevalence of less than 0.18 case per 10,000 population.[2] A few examples of ultra orphan drugs/diseases are[3]
- Imantinib for gastrointestinal stromal tumours.
- Laronidase for mucopolysaccharidosis type 1.
- L-carnitine for genetic carnitine deficiency.
- Anagrelide for essential thrombocytopenia.
- Miglustat for Gaucher's disease.
- Imiglucerase for Gaucher's disease.
- Agalsidase for Fabry's disease.
- Orfadin for hereditary tyrosinaemia type 1.
- Sacrosidase for congenital sucrase isomaltase deficiency.
References
| 1. | Hughes DA, Tunnage B, Yeo ST. Drugs for exceptionally rare diseases: do they deserve special status for funding? QJM 2005;98:829-36. Back to cited text no. 1 [PUBMED] [FULLTEXT] |
| 2. | Rawlins M. Managing rare diseases. London: Royal College of Physicians; 2004. Back to cited text no. 2 |
| 3. | McCabe C, Claxton K, Tsuchiya A. Orphan drugs and the NHS: should we value rarity? BMJ 2005;331:1016-9. Back to cited text no. 3 [PUBMED] [FULLTEXT] |
Copyright 2006 - Indian Journal of Pharmacology
|
