Orphan Drugs in Neurology-A Narrative Review.

ABSTRACT:

Background and aims

Orphan diseases, or rare diseases, are defined in Europe as diseases that affect less than 5 out of every 10,000 citizens. Given the small number of cases and the lack of profit potential, pharmaceutical companies have not invested much in the development of possible treatments. However, over the last few years, new therapies for rare diseases have emerged, giving physicians a chance to offer personalized treatment. With this paper, we aim to present some of the orphan neurological diseases for which new drugs have been developed lately.

Methods

We have conducted a literature review of the papers concerning rare diseases and their treatment, and we have analyzed the existing studies for each orphan drug. For this purpose, we have used the Google Scholar s

SUBMITTER: Sirbu CA 

PROVIDER: S-EPMC10058745 | biostudies-literature | 2023 Feb

REPOSITORIES: biostudies-literature