Management of adults with SMA: Practical considerations and treatment experiences
15 Feb 2024
byProf. Tim Hagenacker, Dr. Shirley Pang
Spinal muscular atrophy (SMA) is an inherited disorder that causes progressive muscle degeneration and weakness. Although there is no cure for SMA, emerging disease-modifying treatments can potentially change disease trajectory and substantially improve patients’ outcomes. At a local symposium, Professor Tim Hagenacker of the Department of Neurology, Essen University Hospital in Essen, Germany, shared his clinical experience and expertise in managing adults with SMA, with a focus on early treatment with an oral drug, risdiplam (Evyrsdi®). Dr Shirley Pang of the Division of Neurology, Queen Mary Hospital, Hong Kong, reported preliminary data on treatment outcomes with risdiplam in Hong Kong.