MUSCULAR ATROPHY, SPINAL

This completed Phase 3 trial tested whether adding the experimental drug apitegromab to standard SMA treatments could improve muscle function in 188 children and young adults (ages 2-21) with later-onset spinal muscular atrophy. All participants were already receiving approved SM…

This study looked at whether adding a drug called nusinersen could help young children with spinal muscular atrophy (SMA) who were still having movement problems even after receiving a one-time gene therapy. Researchers enrolled 46 children under age 3 to receive nusinersen injec…

This study tested a new screening program for babies to detect spinal muscular atrophy (SMA), a severe genetic muscle disease, as early as possible. Over 33,500 newborns in the UK were screened using a tiny extra blood spot from their routine newborn test. The goal was to see if …

This study aimed to understand how well the available treatments work for children with spinal muscular atrophy (SMA) in everyday life, not just in clinical trials. Researchers looked back at the medical records of 213 children in the Czech and Slovak Republics to see their treat…

This study aimed to understand how three different treatments for spinal muscular atrophy (SMA) are actually used in the real world and what they cost the healthcare system. Researchers analyzed anonymous insurance claims data from over 4,100 patients who received one of the trea…

This study looked back at the real-world experiences of 4,805 people with spinal muscular atrophy (SMA) who received one or more of three modern drug treatments. It aimed to understand how often patients needed hospital care or special medical equipment before and after starting …