Lijekovi za rijetke bolesti

Professional paper - Review paper

• Author: Monika Barbarić
• Author: Martina Meštrović
• Author's institution: University of Zagreb; Faculty of Pharmacy and Biochemistry; (Department of medicinal chemistry)
• Scientific / art field, discipline and subdiscipline: BIOMEDICINE AND HEALTHCARE; Pharmacy; Pharmacy

Abstract (english)

There are between 5000 and 7000 rare diseases. These are often chronic, progressive,
degenerative, and often life-threatening diseases. Rare diseases are mostly genetic, and
are therefore present throughout the patient's life, even if symptoms do not appear
immediately. However, many rare diseases appear early in life, and about 30% of
children with rare diseases die before reaching their fifth birthday. There is no cure for
most of the rare diseases, so the art of living with a rare disease is an ongoing learning
experience for patients and families.
»Orphan drugs« are medicinal products intended for the diagnosis, prevention or
treatment of life-threatening or debilitating rare diseases. They are »orphans« because
the pharmaceutical industry, under normal market conditions, has little interest in
developing and marketing drugs intended for only a small number of patients suffering
from very rare conditions.

• Keywords (croatian): rijetke bolesti; lijekovi za rijetke bolesti; orphan drugs
• Language: croatian
• Publication type: Professional paper - Review paper
• Publication status: Published
• Peer review: Peer review - domestic
• Publication version: Published version
• Journal title: Farmaceutski glasnik
• Numbering: vol. 66, no. 6, pp. 325-334
• p-ISSN: 0014-8202
• URN:NBN: urn:nbn:hr:163:588967
• Publication: 2010
• Type of resource: Text
• Access conditions: Open access
• Created on: 2020-09-29 11:19:08