MUSCULAR ATROPHY, SPINAL

This study tested a new medicine called apitegromab in 188 children (ages 2–21) with later-onset spinal muscular atrophy (SMA types 2 and 3) who were already taking standard treatments. The goal was to see if adding apitegromab could further improve muscle function and movement. …

This study looked at whether adding a medicine called nusinersen can help children under 3 with spinal muscular atrophy (SMA) who still have muscle problems even after receiving gene therapy. The 46 children received several doses of nusinersen over about 2 years. Researchers che…

This study looked at whether screening newborn babies for spinal muscular atrophy (SMA) is practical and acceptable to parents in the UK. SMA is a rare genetic disease that destroys nerve cells controlling movement, often leading to severe disability or early death. By testing bl…

This study looked at medical records of 213 children with spinal muscular atrophy (SMA) in the Czech and Slovak Republics. Researchers wanted to see how treatments are actually used in real life and what health outcomes children experience. The goal was to learn more about care p…

This study analyzed insurance claims from over 4,000 people with spinal muscular atrophy (SMA) to see how three approved treatments (Zolgensma, Spinraza, and Evrysdi) are actually used in daily practice. Researchers looked at dosing patterns and healthcare costs over several year…

This study analyzed health records of over 4,800 people with spinal muscular atrophy (SMA) to see how they responded to three different disease-modifying therapies in everyday medical practice. Researchers looked at complications, medical equipment use, and hospital visits before…