Aktuell forskning om Sanfilippos sjukdom – Current Research on Sanfilippo

Impact Report – Cure Sanfilippo Foundation

Impact Report Cure Sanfilippo Foundation

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Save Connor’ Video Raising Funds for First-ever Clinical Trial in Sanfilippo Type C.

New Gene Therapy Candidate for Sanfilippo Type C Ready for Clinical Trial Stage
An investigative gene therapy to treat patients with Sanfilippo syndrome type C was developed at the University of Manchester in England, and Phoenix Nest, a biotech based in the U.S., has signed a licensing agreement to take the therapy to the clinical trial stage.
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Royal Manchester Children’s Hospital
MPS IIIA patient receives world’s first gene therapy treatment for life-limiting genetic condition.
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The gene therapy revolution is here
Gene therapy—for so long something that belonged to the future—has just hit the streets.

You might have picked up a headline alerting us to the most expensive drug in history—a one off gene therapy cure for spinal muscular atrophy. Novartis have priced the drug Zolgensma at A$3 million (US$2.1 million)
Traditionally a parent of a baby with spinal muscular atrophy was told: take your baby home and love her or him. Have no false hope, the baby will die paralyzed and unable to eat or talk by the age of two.
What’s the narrative going to be now? There is a cure but it costs A$3 million.

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