MassBio continued its #Impact2020 Twitter Chat Series with a discussion of rare disease research today and tomorrow, in Massachusetts and beyond.
There are approximately 30 million people living with a rare disease in the USA, between 27 and 36 million in the EU, and collectively around the globe, the rare disease community is estimated to include 350 million people. There are more than 7,000 rare diseases, many of them with no treatment or cure.
Many rare disease therapies are under development in Massachusetts and these leading researchers are pushing for accelerated approval pathways.
In this Twitter chat, we discussed the current state of rare disease research, the clinical and regulatory challenges for rare disease research, and how patients and patient advocates are making a difference.