Unlike any other sector of the pharmaceutical industry, patient groups of #rarediseases are integral to orphan drug development. Traditionally, the strengths of patient groups lay in raising awareness, informing patients and lobbying authorities for access to orphan treatments.
But as lack of treatments for the 8000 rare diseases becomes an increasing concern, patient groups are evolving into “non-profit pharmaceutical companies” and taking on the research responsibilities themselves. Genethon have pioneered in such a manner, specifically the production of gene therapy medicinal products for rare diseases.
To hear from and engage with Genethon and the multitude of other patient groups present at the World Orphan Drug Congress, register now and take advantage of our exclusive early bird offer that expires on the 22nd July!