We are ProQR, a company on a mission to create new medicines for patients in need. We focus on a group of diseases called rare diseases, with a very high need for new medicines. Out of the approximately 7,000 rare diseases, less than 400 have a treatment, and we intend to change that. To treat these diseases we use RNA therapies, a technology that allows us to address inherited diseases that are caused by a genetic defect. Our focus is on diseases that are very severe or life threatening, and have limited treatment options. Amongst others we work on the development of new medicines for several forms of inherited blindness such as Leberâs congenital amaurosis, Usher syndrome type 2 and autosomal dominant retinitis pigmentosa. Source
No articles found.
bluebird bio is pioneering gene therapy with purpose. From our Cambridge, Mass., h...
bluebird bio is pioneering gene therapy with pu...
We make affordable assistive products, starting with a groundbreaking supportive w...
We make affordable assistive products, starting...
We are a gene therapy company dedicated to developing and commercializing novel tr...
We are a gene therapy company dedicated to deve...
At BioMarin, we focus on developing first-in-class and best-in-class therapeutics ...
At BioMarin, we focus on developing first-in-cl...
Novelion Therapeutics Inc. (âNovelion"), headquartered in Vancouver, British Col...
Novelion Therapeutics Inc. (âNovelion"), head...
Arrowhead Pharmaceuticals develops medicines that treat intractable diseases by si...
Arrowhead Pharmaceuticals develops medicines th...
Applied Therapeutics is a clinical-stage biopharmaceutical company developing a pi...
Applied Therapeutics is a clinical-stage biopha...
Join the National Investor Network and get the latest information with your interests in mind.