An Interpretable Data-driven Prediction Model To Anticipate Scoliosis In Spinal Muscular Atrophy In The Era Of (gene-) Therapies

The article discusses the impact of new gene therapies on neuromuscular disorders, specifically 5q-spinal muscular atrophy (SMA). These therapies, including gene therapy and antisense oligonucleotides, have revolutionized the treatment of SMA and have led to improved outcomes for patients. The article also highlights the importance of specialized centers for treating rare diseases like SMA.