Pan-German guidelines on the adverse effects of gene therapy in SMA

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Clinicians from Germany, Austria and German-speaking Switzerland involved in the treatment of SMN1-related proximal spinal muscular atrophy (SMA) have drawn up recommendations concerning the follow-up of children receiving gene therapy and the occurrence of any treatment-related complications:

  • this consensus document, based on a Delphi-style methodological approach, had already resulted in initial recommendations regarding indications for SMA,
  • the 12 consensus proposals set out in this second part focused on the initial diagnostic assessment, safety issues and the management of adverse effects associated with this innovative therapy,
  • with a particular focus on the monitoring of liver transaminases,
  • two further recommendations were made regarding the course of action to be taken in the event of a positive neonatal screening result.

These recommendations were also drawn up in consultation with representatives of patients with SMA.

Delphi consensus on gene therapy of spinal muscular atrophy with onasemnogene abeparvovec in Germany, Austria and Switzerland – part II – expert based recommendations for surveillance and management of side-effects. Ziegler A, Weiß C, Vill K, Baumann M et al. J Neuromuscul Dis. 2026 Aug.

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