Five-year-old boy with Sanfilippo Syndrome faces £3m treatment cost
A mother in Swansea says her five-year-old son with Sanfilippo Syndrome will cost almost £3m for US-approved gene therapy.
Rare condition diagnosis details
Tate McDaid was diagnosed with Sanfilippo Syndrome when he turned four. This rare form of dementia affects just over 100 children in the UK.
Treatment approval and pricing
Health officials approved the first gene therapy for this condition in the US in September. The treatment price is set at $3.95 million per child according to Ultragenyx.
Funding uncertainty for families
Tammy McDaid contacted the drug developer to understand costs for families outside the United States. It remains unclear how UK families will cover this one-time expense without American health insurance.
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