A mom was overjoyed when she learned a gene therapy treatment could save her child's life, until she found out no one would ...

A group of residents in Indore has launched a campaign to raise Rs 9 crore to save the life of a three-year-old girl suffering from a rare genetic disorder called Spinal Muscular Atrophy (SMA) Type 2.

A new review of studies reveals that incorporating SMA into newborn screening programs yields clinical improvements and ...

Determining which patients should receive the treatment will still require individual risk-benefit assessments, experts say.

A new class of life-saving drugs is helping children who once had no hope. But some carry a price tag of millions for a ...

Disability life coaching is turning out to be just the balm columnist Brianna Albers needed to recall the importance of community in SMA.

“Christmas should be full of joy and togetherness, but for some families, they will be spending Christmas in Starship Children’s Hospital,” says Starship Foundation CEO, Jo Simon. “Our campaign is a ...