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Deadly motor-neuron disease treated in the womb in world 1st
An infant with a fatal genetic disease has survived past the age of 2 with no signs of the condition, thanks to treatment ...
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Risdiplam: First In-Utero Treatment for Spinal Muscular Atrophy
Discover the first in-utero treatment for spinal muscular atrophy (SMA) using risdiplam. Promising results show no SMA signs ...
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Spinal Muscular Atrophy Treated in the Womb for the First Time
Spinal muscular atrophy, a progressive neurodegenerative disorder set in motion before birth, could be treated in utero, a ...
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Promising results from first prenatal therapy for spinal muscular atrophy
Spinal muscular atrophy (SMA) is a progressive neurodegenerative disorder set in motion before birth. Scientists at St. Jude ...
This rare genetic disorder was just treated in the womb for the first time ever
Following the first-ever treatment for spinal muscular atrophy in the womb, physicians say a 2-year-old girl shows no signs ...
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FDA approves Evrysdi tablets for spinal muscular atrophy
The FDA has approved Evrysdi in tablet form for patients with spinal muscular atrophy aged 2 years and older who weigh more ...
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First Prenatal Spinal Muscular Atrophy Therapy Shows Promise
February 19, 2025) Spinal muscular atrophy (SMA) is a progressive neurodegenerative disorder set in motion before birth. Scientists ...
Discover Magazine1d
Prenatal Treatment Offers Hope for Infants Born With Spinal Muscular Atrophy
Learn about the promising advance in prenatal treatment of spinal muscular atrophy, introducing a potential approach to suppress symptoms of the disorder in infants.
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Roche’s Evrysdi Becomes First Pill Cleared for Spinal Muscular Atrophy
The FDA on Wednesday approved the industry’s first-ever tablet treatment for spinal muscular atrophy— Roche ’s SMN2 splicing ...