A new nonprofit wants to streamline gene therapy for diseases often avoided by pharmaceutical companies — making treatment more like a routine procedure than a bespoke drug.
Researchers have made important strides in understanding and treating diffuse midline gliomas, which were long seen as a death sentence for the children and adolescents they affect.
In a Wednesday news release, the FDA announced it had approved Casgevy, the first gene therapy for children with sickle cell disease.
The world's first human trial of whether a drug can essentially make a person's cells younger sets up a reality check for one of the longevity field's most promising theories. Why it matters: The early-stage clinical trial will signal whether "cellular reprogramming" can be performed safely in humans, a prerequisite for any future claim that a treatment can slow or even reverse biological aging. The big picture: Attempts to prolong the human lifespan can refer to anything from better diet and exercise to the infusion of blood plasma from young donors. - The former is validated by decades of science; the latter has merited two warnings from the Food and Drug Administration that said such treatments lack any evidence of clinical benefit. …
The breakthrough could one day allow doctors to repair disease-causing mutations in embryos.
Decades of scientific investment have paid off just in the last month, with researchers announcing promising breakthroughs against cancers and other deadly afflictions. Why it matters: They're all the product of a rigorous innovation system that delivers — though some have one important limitation: All they can do is buy someone a few more years. The big picture: We're still in the era of medicine where most miraculous new drugs don't cure the disease as much as allow sick people to live longer. - In economic terms, that ongoing medical care is expensive, and long-term diseases usually limit the number of years people are productive while increasing their lifetime medical costs. Driving the news: Recent weeks have featured a series of…
The Allen Institute in Seattle says scientists have now learned enough about how the brain works to start fixing it when it breaks.
The treatment, developed by Regeneron Pharmaceuticals, is for a very rare form of deafness. But it represents a medical milestone.
The treatment, the first of its kind, was approved by the Food and Drug Administration on Thursday. “Our baby was born deaf, and now he can hear,” said one parent.
We can’t let the most important medical achievement of a decade slip through our fingers.