What happens when a $2 million gene therapy is not enough

By Deena Beasley LOS ANGELES (Reuters) - Baby Ben Kutschke was diagnosed at three months with spinal muscular atrophy, a rare inherited disorder which is the leading genetic cause of death in infancy globally. It leaves children too weak to walk, talk, swallow or even breathe. So when in 2021 his parents heard about Zolgensma – a one-time therapy costing millions of dollars that promises to replace genes needed for the body to control muscles – they had high hopes. They were disappointed. After treatment with the $2.25 million therapy at almost eight months old, Ben was able to hold his head u...