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A small report about patisiran

Léo Sumi

1 June 2019

I had lectures on pharmacology (BIO-478) during my studies at EPFL. A small personal project was done for this course. Here, you will find the final report submitted on 17 May 2019 and accepted on 21 May 2019.

The report talk about patisiran, a new approved drug in 2018 and commercialized under the name OnpattroTM. The drug is used against hereditary transthyretin amyloidosis, a progressive and life-threatening disease.

Be aware that it is not the easiest text to read if you do not have some knowledge on the subject. Also, be aware that the text simplify lots of mechanisms. If you want a more complete description, you can take a look at the review from Setten et al. for instance.

The report is called RNA interference therapy with patisiran for hereditary transthyretin amyloidosis. The PDF can be found here.