Results from a Phase 3 clinical trial evaluating the use of high-dose genistein in children with Sanfilippo syndrome shows that its use has no meaningful clinical benefit. Sanfilippo syndrome — also known as Mucopolysaccharidosis III — encompasses a group of rare diseases that are caused by a deficiency in one…
New formulations based on sugar molecules may improve the properties of candidate compounds for substrate reduction therapy to treat Sanfilippo syndrome, researchers have found. The study, “Cyclodextrin encapsulation of daidzein and genistein by grinding: implication on the glycosaminoglycan accumulation in mucopolysaccharidosis type II…
Substrate reduction therapy (SRT) shows potential in preclinical models and in clinical studies of patients with Sanfilippo syndrome. Treatment with genistein, currently being studied a Phase 3 trial, is the most promising candidate so far, according to a recent review. The review,…
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