It has raised broader questions around high-cost gene therapies entering the market. Read more at straitstimes.com.
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.
South Africa Latest News, South Africa Headlines
Similar News:You can also read news stories similar to this one that we have collected from other news sources.
Book review: Zero Days is a perfectly thrilling beach readRuth Ware's latest offers action and a strong female protagonist. Read more at straitstimes.com.
Read more »
The Big Read: First-time voters can play key role in deciding presidential race — do they care enough?2/ AT A GLANCE: 🗳️Are you a first time Presidential Election voter? Most first-time PE voters whom TODAY spoke to have limited knowledge of or wish they know more about the President’s role. Here are some examples of what the President can and cannot do.
Read more »
Maliki visits vegetable farm and polytechnic during three-day visit to Riau IslandsHe attended a National Day reception and played the guzheng to raise funds for Red Cross Batam. Read more at straitstimes.com.
Read more »
Tourists asked to leave Hawaii’s Maui as wildfires burnBefore the wildfires, the tourism economy was recovering from a pandemic-induced visitor slump. Read more at straitstimes.com.
Read more »
Philippine military says will send more supplies to disputed reefThe delivery could take place as early as next week. Read more at straitstimes.com.
Read more »