Spinal Muscular Atrophy: A Rare but Treatable Disease of the Nervous System

James N. Sleigh, Vanessa Christie-Brown, Liz Ryburn, R. J. Yáñez-Muñoz

Research output: Contribution to journalReview articlepeer-review


When something is rare it means that it happens very infrequently. Did you know that most diseases are rare? There are more than 6,000 known rare diseases, each affecting fewer than 1 in every 2,000 people. But if we put all the rare diseases together, they affect about 1 in 17 of us! Given that they are individually uncommon, rare diseases are often poorly understood. However, rare diseases have a large impact on families and society, thus they require increased attention. In this article, we will explore a rare disease of the nervous system called spinal muscular atrophy (SMA). We will tell you about the symptoms of SMA and explain how it is inherited. SMA has led the way in the discovery of treatments for rare diseases. Finding treatments for rare diseases requires intensive research and commitment from many people, but the success of SMA treatments highlights the importance of studying other rare conditions.
Original languageEnglish
JournalFrontiers for Young Minds
Publication statusPublished - 1 Aug 2023

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