Short-term safety results from compassionate use of risdiplam in patients with spinal muscular atrophy in Germany.
Andreas HahnRené GüntherAlbert LudolphOliver SchwartzRegina TrollmannPatrick WeydtMarkus WeilerKathrin NeulandMartin Sebastian SchwadererTim Hagenackernull nullPublished in: Orphanet journal of rare diseases (2022)
We present the first real-world safety data of risdiplam in patients with SMA in Germany. Our observations indicated no new safety signals under real-world conditions. Real-world SMA1/2 populations comprise considerable numbers of patients who are not eligible for gene therapy and cannot tolerate or have failed nusinersen treatment. This medical need may be addressed by oral risdiplam.