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Current Medicinal Chemistry


ISSN (Print): 0929-8673
ISSN (Online): 1875-533X

Review Article

Repositioning Drugs for Rare Immune Diseases: Hopes and Challenges for a Precision Medicine

Author(s): Erica Valencic, Alenka Smid, Ziga Jakopin, Alberto Tommasini and Irena Mlinaric-Rascan*

Volume 25, Issue 24, 2018

Page: [2764 - 2782] Pages: 19

DOI: 10.2174/0929867324666170830101215

Price: $65


Human primary immunodeficiency diseases (PIDs) are a large group of rare diseases and are characterized by a great genetic and phenotypic heterogeneity. A large subset of PIDs is genetically defined, which has a crucial impact for the understanding of the molecular basis of disease and the development of precision medicine.

Discovery and development of new therapies for rare diseases has long been de-privileged due to the length and cost of the processes involved. Interest has increased due to stimulatory regulatory and supportive reimbursement environments enabling viable business models.

Advancements in biomedical and computational sciences enable the development of rational, designed approaches for identification of novel indications of already approved drugs allowing faster delivery of new medicines. Drug repositioning is based either on clinical analogies of diseases or on understanding of the molecular mode of drug action and mechanisms of the disease. All of these are the basis for the development of precision medicine.

Keywords: Drug repositioning, repositioning, primary immunodeficiency diseases; pediatrics, new therapies, rare diseases.

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