Solid Biosciences‘ experimental gene therapy for Friedreich’s ataxia (FA), called SGT-212, has been granted orphan drug status in the European Union. This designation aims to offer additional incentives to companies investing in treatments for rare diseases, where the small number of patients can make it hard for drug…

Last weekend, my mother, daughter, and I went to a glass-blowing studio to make decorative glass pumpkins. It was such a fun experience, and we learned so much about the 2,000-year-old art of glass blowing. I am now fully dependent on a wheelchair due to my Friedreich’s ataxia (FA)…

Growing up, Halloween was always one of my favorite holidays. My birthday falls a couple of weeks after the holiday, so I always combined the two. For years, I threw Halloween-themed birthday parties, complete with costumes, games, and, most importantly, cookie cake. Each year, we gathered in a local restaurant’s…

Using an experimental gene-editing approach, a team of U.S. scientists was able to successfully and precisely remove the genetic defect underlying most cases of Friedreich’s ataxia (FA) in several laboratory cell models. According to the researchers, the strategy achieved “precise editing” — as high as 98.2% — among connective…

Last week, I sat with my service dog near my home’s entrance, waiting for public transportation to take me to a doctor appointment. It was a cool fall day with drizzling rain — the kind of day that made me want to cancel the appointment and cozy up by the…