"We're ready, and these children are waiting..." Please reach out if you can help!
CEO, Founder | Biotech, Healthtech | Board Member, Advisor | Speaker, Advocate for Health Innovation | Rare Disease
This is back-to-school week, a joyful moment many rare disease families don’t get to experience. Of the 150 million+ children worldwide with #rarediseases, 50 million won’t live to their first day of kindergarten. Millions more will be unable to speak, walk or achieve other milestones. Sadly, the science is out there now to help many of these kids. As we’ve worked to establish the Orphan Therapeutics Accelerator, we’ve come across dozens of #treatments shown to be safe and potentially effective (even curative) in clinical trials, which companies can’t (lacking funds) or won’t (not commercially lucrative enough) move forward. For a small fraction of what is being invested in chronic diseases (a $1bn deal was announced yesterday for yet another #obesity drug) or other condition areas, we could complete development of 10-20 of these shelved rare disease treatments within a few years - enabling tens of thousands of children that wouldn’t have otherwise to experience their first day of school. If you are interested in funding or partnering with us, contributing a therapeutic program, or know of others that might be, please reach out. We’re ready, and these children are waiting. John F. Crowley Tim Hunt Sue Kahn Chris Garabedian Walter Kowtoniuk Melissa Stevens Orly Mishan Devin Rosenthal, PhD, MBA Jim Geraghty Giacomo Chiesi Dr. Jeremy M. Levin Tania Simoncelli Priscilla Chan Belinda Termeer Adam Knight, PhD Catriona Crombie Chris Coburn Albert Seymour Adama S. Juan Carlos Lopez Jason P. Hafler Burcu Eryilmaz Bruce Bloom Bruce Goldsmith Christopher Viehbacher Rachelle Jacques Bradley Campbell Thomas Mathers Elizabeth White Nandan Padukone Deanna Portero Gerald Cox