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.
Sanfilippo Syndrome Patients, Drug Developers At Odds With US FDA Guidance
Agency recommendation for randomized controlled trials in ultra-rare pediatric disease is unworkable for various reasons, say advocates and sponsors who seek use of the accelerated approval pathway based on changes in heparan sulfate levels.
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