Gene Therapy: Hope and Hurdles

This chapter explores the advancements and challenges in genetic therapies for children with rare conditions, featuring a parent's journey to advocate for personalized treatments. It discusses the financial and logistical difficulties in developing these therapies, as well as the unconventional fundraising efforts by families in need. The emotional toll on families, particularly the Lockguard family facing the rare condition SBT50, illustrates the urgent need for effective solutions amidst the complexities of the biotech landscape.