Chief Operating Officer @ Inozyme Pharma | Committed to the development and commercialization of innovative therapies for rare and hard to treat diseases.
Last week I was on Capitol Hill advocating for the Priority Review Voucher (PRV) program—a critical initiative that offers hope to children living with rare diseases. Since its inception, the PRV program has transformed the landscape of rare disease treatment, benefiting over 200,000 patients and addressing urgent unmet needs. With ~95% of rare diseases lacking approved treatments, the PRV program incentivizes biopharmaceutical companies to accelerate the development of pediatric rare disease drugs by offering priority reviews from the FDA. However, without reauthorization, this program will expire on September 30, threatening the progress that has been made. At Inozyme Pharma, we are developing novel therapeutics for devastating rare diseases like #ENPP1 Deficiency and #ABCC6 Deficiency, which impact infants, children, and adults. Our mission is driven by the resilience and hope of the families we serve, and the PRV program is vital in helping us bring life-saving treatments to them. Please read my opinion piece on this critical topic published today in BioSpace https://bit.ly/4f14JMG Let's work together to keep innovation alive for millions of children and help ensure a future where no child's potential is diminished by a rare disease. #RareDisease #Advocacy #InozymePharma #CapitolHill #Community #GACI #BioSpace #RDCC
Great efforts Matt, enjoyed the article.
Keep up the great work, Matt! What you and others in the industry are doing is so important for the #raredisease community!
Clinical Project Manager at PDC CRO
1moGreat Job