You've Got a Friend in Me: A Case for Cross-SIG Collaboration
Julien Heidt, Jie (Jenni) Li, Tara MacCannell, Daniel Rosenberg, Timothy Savage, Delphine Saragoussi
There are approximately 7,000 rare diseases which collectively impact more than 250 million people globally (US FDA, 2019) (Wakap et al., 2020). Approximately 80% of rare diseases are associated with a single gene mutation, and about half of these diseases affect children (Batshaw et al., 2014). Many of these rare diseases are life-threatening, and most have no approved disease-modifying therapies, representing substantial unmet medical need among pediatric patient populations. Recognizing this gap, researchers and manufacturers are making strides in the development of new therapies. Continue reading.
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