We ask the Government to urgently fund rebisufligene etisparvovec-hopf for eligible children with Sanfilippo syndrome Type A, working with relevant authorities and manufacturers to establish the fastest possible route to treatment.
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Sanfilippo Type A is a rare, progressive childhood dementia. UK children currently cannot access rebisufligene etisparvovec-hopf for their treatment. We are asking the Government to urgently provide funding to allow eligible children to access this treatment. Sanfilippo causes irreversible loss of skills, so families cannot afford years of delay. Every day matters.