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.
Ben is one of a growing number of patients with spinal muscular atrophy whose doctors are turning to additional drugs on top of the gene therapy, six top US neurologists told Reuters. Other SMA therapies need to be taken continuously, but Zolgensma’s price was justified by hopes this revolutionary approach could beat the condition once and for all.
Baby Ben Kutschke was diagnosed at three months with spinal muscular atrophy, a rare inherited disorder. PHOTO: REUTERS “The perception that Zolgensma is going to be a complete cure ... is not coming to fruition from the data we have seen over the last four years,” said Dr. Roger Hajjar, director of the Mass General Brigham Gene & Cell Therapy Institute.
“People still have misconceptions about Zolgensma,” Ms Kutschke told Reuters. “It is a treatment, not a cure.
The economics of treating the relatively small number of patients make a high price tag imperative for manufacturers.