Murdoch Children’s Research Institute advances KAND treatments as ASO therapy shows promise
Updated
Updated · Fox News · May 5
Murdoch Children’s Research Institute advances KAND treatments as ASO therapy shows promise
5 articles · Updated · Fox News · May 5
Founder Luke Rosen said his daughter Susannah, diagnosed at two, was among the first patients to receive antisense oligonucleotide therapy through the Australian institute’s work.
Her family reported meaningful improvements, while researchers pursue gene therapy, traditional drug development and other approaches in parallel to speed potential treatments for the rare neurodegenerative disorder.
KAND causes seizures and progressive loss of cognition, motor skills and vision, has no cure, and the treatment is not yet approved in Australia, leaving families seeking broader access.
When experimental gene therapies carry fatal risks, how do families and doctors decide if a potential cure is worth the gamble?
As personalized medicine creates million-dollar cures, how do we prevent a future where only the wealthy can afford a chance to live?