Ewout Groen @ewout.bsky.social · 13/11/2024Our findings highlight that despite understanding genetic causes and having treatments available, optimal treatment of genetic diseases remains challenging. Exciting to see all the insights the modest fibroblast could give us 🔬🧫 030
Ewout Groen @ewout.bsky.social · 13/11/2024We found that in 57% of cases, cells showed a significantly better response to either nusinersen or risdiplam, despite similar mechanisms. This suggests treatment effectiveness varies between patients more than previously thought. 2/3 110