Many patients with spinal muscular atrophy (SMA) are currently relying on recently approved SMA-specific treatments. However, a majority of patients do not feel controlled under their actual treatment plan and have high expectations of future treatment breakthroughs, according to a recent press release.

According to recent statistics, there are approximately 25,000 patients with SMA in the United States (US). More than 50% of patients require 24-hour care and the majority of them require assisted breathing, wheelchairs, or both. The average age of these patients is 40; over 90% suffer from other comorbidities such as anxiety and scoliosis, and over half of them have an income below $55,000 per year.

In the last decade, the FDA has approved 3 SMA-specific treatments based on gene therapy. Approximately 90% of US patients included in the study are currently receiving or have received one of these treatments. Nonetheless, experiences vary greatly among patients; more than 70% of patients do not feel their symptoms are currently controlled and fear disease progression, and 20% of them plan to request a treatment change from their attending physician.

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Unsurprisingly, the majority of patients included in the survey intend to seek new treatments. Only new assistive devices with the capacity to improve quality of life outscore new SMA-specific treatments in the top priorities of patients with SMA.

More than 40% of patients believe monetary issues prevent them from receiving all the care they need. However, 44% are certain that they are currently taking all necessary measures to correctly manage their condition.

Regarding access to care, although the majority of patients have access to a neurologist, over 60% identify internists or family physicians as their main healthcare providers. Yet, approximately half of patients report not having access to an SMA specialist and deny knowing how to gain access.

“With a number of recently approved treatments already having an impact on quality of life and an intriguing treatment pipeline, people with spinal muscular atrophy are actively looking for accurate information, including the treatment experiences of others,” said Olivier Chateau, Health Union’s co-founder and chief executive officer.


SMA survey findings: SMN-enhancing gene therapies being used, having impact. News release. Health Union. October 18, 2022.