An infant with a fatal genetic disease has survived past the age of 2 with no signs of the condition, thanks to treatment ...

Spinal muscular atrophy (SMA) is a debilitating genetic condition that’s usually fatal by a few years of age. But an ...

The mother took the drug daily for six weeks while the baby herself was given the drug orally, roughly one week after the ...

Looking for the perfect gift for a newborn? Our guide to the best newborn gift packs features premium, eco-friendly, and ...

Rett syndrome is a rare genetic disease affecting 1 in 10,000 newborn girls, characterized by sudden regression around 1 year ...

Scientists report results from a promising new approach to treat the rare neurodegenerative disorder. Spinal muscular atrophy (SMA) is a progressive neurodegenerative disorder set in motion before ...

A new study will investigate whether prenatal antibiotic use impairs breast milk immune function and disrupts neonatal gut ...

Discover the first in-utero treatment for spinal muscular atrophy (SMA) using risdiplam. Promising results show no SMA signs ...

Spinal muscular atrophy, a progressive neurodegenerative disorder set in motion before birth, could be treated in utero, a ...

Researchers at the Josep Carreras Leukaemia Research Institute revealed a new aspect of Rett Syndrome’s pathogenesis that can ...

Spinal muscular atrophy (SMA) is a progressive neurodegenerative disorder set in motion before birth. Scientists at St. Jude Children's Research Hospital led the first in uterotreatment of SMA with ...