Spinal muscular atrophy (SMA) is a rare hereditary disorder that causes progressive damage to the neuromuscular system, resulting in muscle wasting and weakness. Anna Kostera-Pruszczyk, MD, PhD, Medical University of Warsaw, Warsaw, Poland, comments on the changing landscape of SMA, which has been revolutionized in recent years with the introduction of several efficacious disease modifying therapies. Nusinersen was approved by the EMA for use in pediatric and adult patients in 2017. More recently in 2021, risdiplam was also approved for patients 2 months of age and older. These agents have been shown to not only prevent disease progression, but aid in motor function restoration. Prof. Kostera-Pruszczyk highlights the challenges facing adult patients and the need for more data on the natural history of SMA, to help inform patients on what they can expect from their treatment. This interview took place at the European Academy of Neurology (EAN) 2022 Congress in Vienna, Austria.
