Patient advocates, sponsors and clinical experts are protesting what they view as generally unworkable recommendations from the US Food and Drug Administration on development approaches and clinical trial design for new therapeutics for Sanfilippo syndrome.
Key Takeaways
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FDA’s 2020 draft guidance on development of treatments for MPS III recommends randomized controlled trials, which disease community representatives say are infeasible.
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Advocates and...
Sanfilippo syndrome, also known as mucopolysaccharidosis type III (MPS III), is an ultra-rare enzyme defect disease that affects young children. It was the focus of an FDA patient listening session...
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