Today we are taking a look at guidelines related to Spinal Muscular Atrophy (SMA). Through the following timeline we will see how these recommendations have evolved in response to advances in our understanding of the disease’s properties and the emergence of new interventions.

Spinal Muscular Atrophy Timeline
  • 2005  – A committee of international healthcare and SMA experts, the International Standard of Care Committee for Spinal Muscular Atrophy, convened to plan guidelines for SMA and generate consensus statements regarding standards of care.
  • 2016 – Spinraza (nusinersen) was approved by the U.S. FDA for treatment of SMA in children and adults. 
  • 2017 – 2017 International Standards of Care for SMA was published following the approval of Spinraza. This was a two-part update to the previous 2007 version:
    • Part 1 focused on recommendations for diagnosis, rehabilitation, and orthopedic and nutritional care.
    • Part 2 focused on recommendations for pulmonary and acute care, medication, supplements, immunizations, other organ systems, and ethics.
  • 2019 – Zolgensma (onasemnogene abeparvovec-xioi) approved by the U.S. FDA as a one-time infusion for children younger than 2 with SMA.
  • 2020 – Evrysdi (risdiplam) approved by the U.S. FDA to treat SMA in children and adults.
  • 2024 – The standards of care were updated in 2024 based on the latest SMA treatment approvals and research.

With three approved treatments for SMA, and the latest standards of care updated to include the three approved treatments, that brings us up through today.

Looking ahead, there are a handful of ongoing clinical trials evaluating the safety and efficacy of new SMA treatment options. Let’s take a look at a few.

Ongoing Clinical Trials for SMA Treatment

A Study to Evaluate the Efficacy and Safety of Taldefgrobep Alfa in Participants with Spinal Muscular Atrophy

A Study to Investigate the Safety and Efficacy of RO7204239 in Combination with Risdiplam (RO7034067) in Participants with Spinal Muscular Atrophy

Long-term Safety & Efficacy of Apitegromab in Patients with SMA Who Completed Previous Trials of Apitegromab

Long-term Follow-up of Patients with Spinal Muscular Atrophy Treated with OAV101 in Clinical Trials

With these, and many more, treatment options actively being investigated, there will likely be additional updates to the SMA standards of care as new, successful options are determined.

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