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5:18
stemcellthailand.org
Stem Cell Treatment for SMA - Spinal Muscular Atrophy
Neural stem cell transplants for SMA and gene therapy are the most effective treatment options in 2025 for control and remission of spinal muscular atrophy.
Jan 16, 2025
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321K views · 2.4K reactions | Today, the Food and Drug Administration approved a new gene therapy for Spinal Muscular Atrophy. SMA Type 1 is the number one genetic killer of children 2 and under. This first-time gene therapy, developed here at Nationwide Children's, is allowing kids like Evelyn to have a bright future. Watch her incredible story. | Nationwide Children's Hospital | Facebook
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🧠 Understanding Spinal Muscular Atrophy (SMA)🧠 Spinal Muscular Atrophy is a genetic disorder that affects the motor neurons in the spinal cord, leading to muscle weakness and atrophy. Here’s what you need to know: What is SMA? - Definition: A group of inherited disorders characterized by the degeneration of motor neurons, which are vital for muscle control. - Symptoms: Includes muscle weakness, difficulty in movement, and in severe cases, respiratory issues. Who is at Risk? - Genetics:Individu
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