Despite significant progress made in research and development in recent decades, it remains a fact that there are no FDA-approved therapies for more than 90% of the approximately 7,000 rare diseases that impact hundreds of millions of people worldwide.
In this article published in PharmaVoice, Pete Robinson and Ray Huml, heads of the Syneos Health Cell & Gene Therapy and Rare Disease Consortia respectively, provide their commentary on how the industry can overcome the hurdles to bringing innovative therapies to the rare disease market.
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