Families Advocate for Gene Therapy Cures

The recent trend of biotech companies halting the development of gene therapies raises concerns about the viability of such treatments for rare diseases, with over 50 therapies abandoned since 2021, prompting parents and scientists to advocate for these stalled projects. Nicole Johnson's efforts to find a cure for her daughter’s FOXG1 syndrome exemplify the urgency in this field. In a separate story, a Louisiana nurse, Morgan Rachal, discovered her daughter's potential Sanfilippo Syndrome through a TikTok video, leading to an early diagnosis that may help mitigate irreversible damage. Meanwhile, Lisa Whitehead, a breast cancer survivor, is organizing a fundraiser to support Breast Cancer Trials, which has conducted numerous clinical trials in search of better treatments. These narratives highlight the ongoing battles faced by families grappling with rare diseases while underscoring the importance of continued research and funding for effective treatments. Overall, these stories reflect a broader concern regarding the pace of medical advancements in treating severe and rare conditions.