What happens when a $3m gene therapy is not enough
LOS ANGELES - 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...