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  • Format: ePub

Spinal Muscular Atrophy (SMA) is a rare but devastating genetic disorder that affects motor neurons, leading to progressive muscle weakness and, in severe cases, death. The condition is caused by the loss of motor neurons in the spinal cord, resulting in a lack of signals being transmitted to the muscles. This results in difficulty with essential motor functions, such as walking, breathing, and swallowing. SMA primarily affects infants and children, though adults may also be diagnosed with a milder form of the disease.
Until recently, SMA was considered a fatal disorder with limited
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Produktbeschreibung
Spinal Muscular Atrophy (SMA) is a rare but devastating genetic disorder that affects motor neurons, leading to progressive muscle weakness and, in severe cases, death. The condition is caused by the loss of motor neurons in the spinal cord, resulting in a lack of signals being transmitted to the muscles. This results in difficulty with essential motor functions, such as walking, breathing, and swallowing. SMA primarily affects infants and children, though adults may also be diagnosed with a milder form of the disease.

Until recently, SMA was considered a fatal disorder with limited treatment options, primarily focusing on managing symptoms rather than addressing the root cause. However, in the past decade, advancements in gene therapy have significantly transformed the prognosis for individuals with SMA. Groundbreaking treatments are now available, offering hope for families affected by this condition.

This e-book explores the complexities of Spinal Muscular Atrophy, including its causes, symptoms, and classifications, as well as the latest gene therapies that are changing the landscape of treatment. We will delve into the science behind these therapies, their clinical efficacy, and the future of SMA treatment. Through this guide, readers will gain a comprehensive understanding of SMA and the revolutionary gene therapies that hold the potential to transform the lives of patients worldwide.


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