A new Science Battle for Wingspan...
The extraordinary story of Emily Kramer-Golinkoff’s fight against cystic fibrosis (CF) and her urgent mission to secure breakthrough genetic therapies for the estimated 10% of patients who cannot benefit from existing treatments.
Diagnosed with CF at just six weeks old, Emily spent her childhood managing the disease through intensive daily treatments. Despite her efforts, by college her lung function had fallen to around 30%. Around the same time, a revolutionary new class of drugs called CFTR modulators became available, but they couldn’t help Emily because her rare genetic mutation means she produces no CFTR protein for the drugs to repair.
Devastated but refusing to give up, in 2011 Emily and her family founded Emily's Entourage, a nonprofit dedicated to funding research and development of new treatments for CF that replace or repair the CF gene, raising over $20 million in the last 15 years.
The film documents Emily’s fight and showcases the work of leading CF scientists who specialise in CF genetic therapies – including DNA gene therapy, mRNA therapy and gene editing. These not only offer hope for ‘the 10%’, but may in future lead to a genetic cure for CF.