New therapies for spinal muscular atrophy: where we stand and what is next

Research output: Contribution to journalArticle


The natural history of spinal muscular atrophy has been radically changed by the advent of improved standards of care and the availability of disease-modifying therapies. The aim of this paper is to provide the current therapeutic scenario including new perspectives and to report the challenges related to new phenotypes a few years after the therapies have become available. The paper also includes a review of real-world data that provides information on safety and efficacy in individuals that were not included in clinical trials. Special attention is paid to future perspectives both in terms of new drugs that are currently investigated in clinical trials or providing details on current developments in the use of the available drugs, including combination therapies or new modalities of dose or administration.Conclusion: Clinical trials and real world data support the efficacy and safety profiles of the available drugs. At the moment there is not enough published evidence about the superiority of one product compared to the others.
Original languageEnglish
Pages (from-to)N/A-N/A
JournalEuropean Journal of Pediatrics
Publication statusPublished - 2023


  • Clinical trial
  • Combined therapies
  • Therapy
  • Spinal muscular atrophy
  • Real-world data


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