För information om aktuell forskning och studier tipsar vi om att kontakta eller läsa mer på nedan länkar:
- Cure Sanfilippo Foundation
- Sanfilippo Children’s foundation
- Phoenix Nest Inc
- Lysosomal Disease Network
- Brain – a journal of neurology
- Clinical trial programs for Sanfilippo (MPS III)
- Svenska MPS-föreningen
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.