New gene therapy corrects a form of inherited macular degeneration in canine model
(University of Pennsylvania) Researchers from the University of Pennsylvania have developed a gene therapy that successfully treats a form of macular degeneration in a canine model. The work sets the stage for translating the findings into a human therapy for an inherited disease that results in a progressive loss of central vision and which is currently untreatable.
US approves the one-time treatment for deadly spinal muscular atrophy in infantsSwiss drugmaker Novartis has received US approval for itsspinal muscular atrophy gene therapy Zolgensma – pricing the one-time treatment at a record $2.125m.The Food and Drug Administration on Friday approved Zolgensma for children under the age of two with SMA, including those not yet showing symptoms. The approval covers babies with the deadliest form of the inherited disease as well as those with types where debilitating symptoms may set in later.Continue reading...
The price set by the Swiss drugmaker Novartis may be the world ’s highest for a single treatment — prompting renewed debate about how society will pay for gene-therapy breakthroughs.
Swiss drugmaker Novartis on Friday won U.S. approval for its gene therapy Zolgensma for spinal muscular atrophy (SMA), the leading genetic cause of death in infants, and priced the one-time treatment at a record $2.125 million.
When Malachi Anderson was diagnosed with a rare and often deadly disease called spinal muscular atrophy (SMA) as an infant nearly four years ago, his parents Tina and Torence had a decision to make.
U.S. regulators have approved the most expensive medicine ever, a $2.125 million gene therapy meant to cure a disorder that rapidly destroys a baby's muscle control and kills most within a couple years
The Food and Drug Administration on Friday approved a $2.125 million treatment for spinal muscular atrophy, a genetic illness known as muscle-wasting disease that kills many babies before they turn 2.
A single, one-time infusion of onasemnogene abeparvovec-xioi (Zolgensma) can minimize the progression of SMA and improve survival in children younger than age 2 years — and will cost over $2 million.FDA Approvals
At $2,125,000, it is the most expensive drug in the world
U.S. regulators approve gene therapy priced at $2.125 million US to treat a rare condition called spinal muscular atrophy.
(CNN) — The US Food and Drug Administration approved a treatment Friday for a genetic disease called spinal muscular atrophy that causes infants’ muscles to waste away, potentially killing them before age 2. And then came the price tag: $2.125 million for a one-time treatment. The gene therapy, called Zolgensma, will be marketed by AveXis, whose parent company is Novartis. “Today’s approval marks another milestone in the transformational power of gene and cell therapies to treat a wide range of diseases,” Dr. Ned Sharpless, the FDA’s acting commissioner, said in a statement Friday. &ldqu...