
Race Against Time to Fund Life-Saving Sanfilippo Treatment for a Toddler
Two-year-old Leni Forrester’s Sanfilippo syndrome progression could be halted by a US clinical trial, but UK funding is lacking. Her parents are urging government support and newborn screening expansion while raising funds (the trial costs about £5.5m) to access the treatment before irreversible brain damage and loss of mobility or speech occur.





