First-Ever Prenatal Treatment for Spinal Muscular Atrophy Shows Promise

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Promising results from first prenatal therapy for spinal muscular atrophy

Scientists report results from a promising new approach to treat the rare neurodegenerative disorder.

MedPage Today

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First-Ever Prenatal Treatment for Spinal Muscular Atrophy Shows Promise

(MedPage Today) -- The first prenatal therapy for spinal muscular atrophy (SMA) showed promising results, a case report indicated. More than 2 years after the child was born, no identifiable features of SMA have been observed, reported Richard...

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A girl in the U.S. shows no signs of spinal muscle atrophy after she has been given the drug risdiplam in the womb.

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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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In single case, spinal muscular atrophy drug given before birth spared baby of genetic disease symptoms

A mother took the spinal muscular atrophy drug Evrysdi before giving birth to a baby with the devastating genetic disease. The baby, who went on to receive gene therapy months before they turned two, has ...