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04.03.2025

Breakthrough in Spinal Muscular Atrophy Treatment: Hope for a Future Without the Condition

A two-and-a-half-year-old girl has shown no signs of spinal muscular atrophy, a rare genetic disorder, after being treated with a gene-targeting drug while in the womb. The child’s mother took the drug during late pregnancy, and the child continues to take it. This breakthrough treatment has the potential to change the lives of thousands of children born with the condition.

Forecast for 6 months: Expect an increase in research and development of gene-targeting treatments for spinal muscular atrophy, with several new clinical trials announced in the next six months.
Forecast for 1 year: By the end of the year, expect the FDA to approve more gene-targeting treatments for spinal muscular atrophy, paving the way for widespread adoption and improved patient outcomes.
Forecast for 5 years: In the next five years, expect a significant reduction in the number of children born with spinal muscular atrophy, thanks to the widespread adoption of prenatal testing and gene-targeting treatments.
Forecast for 10 years: By the end of the decade, expect spinal muscular atrophy to be largely eradicated, with the condition becoming a rare occurrence in developed countries.

Tags: development, DNA genome genetic, health, research