Tough Genes

Tough Genes, along with over 200 other organizations, proudly signed a joint letter calling Congress to approve a long-term extension of the Priority Review Voucher program through the Creating Hope Reauthorization Act. What does this mean and why is this important to us? Developing therapeutic drug treatments for rare pediatric disorders is challenging. Faced with small patient populations, delays in diagnosis and difficulties conducting clinical trials for children, the average time the FDA takes to approve a drug for use is over 15 years. According to the EveryLife Foundation, a cowriter for the letter, “49 Rare Pediatric PRVs have been issued since 2012 for innovative treatments in 40 diseases like spinal muscular atrophy, Duchenne muscular dystrophy, and progeria syndrome. 7 out of 7 drug developers interviewed by the GAO reported that PRVs were a factor in drug development decisions.” What this means is, thanks to PRVs, lifesaving, or life enhancing therapeutic drugs have been made available for 40 diseases and countless patients. Over the past decade, the Rare Pediatric Disease (RPD) Priority Review Voucher (PRV) program has been an invaluable tool, expediting new treatments for children with rare conditions and genetic disorders just like IRF2BPL-Related Disorder. We thank the EveryLife Foundation and the National Organization for Rare Disorders for collaborating, writing and distributing this letter.

Jeans for Genes Week 2024 By wearing your favorite jeans, you can help raise awareness and funds for children affected by life-altering genetic conditions like IRF2BPL-Related Disorder. 💙👖 Send in your pictures and we will feature them on our social media pages throughout the week! Don’t forget to donate! Every contribution brings us one step closer to finding a treatment. To learn more or donate, visit www.toughgenes.org

Tough Genes is on a mission to find a treatment for IRF2BPL-Related Disorder. We are dedicated to advancing scientific knowledge and medical understanding of the IRF2BPL gene. Please consider donating today through the below link or our website. Your generous contribution is so appreciated! https://lnkd.in/gFBJrjyd Learn more at www.toughgenes.org