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Experts Release Recommendations for Future Sanfilippo Syndrome Trials

A group of experts has discussed and released a series of recommendations for future trials’ design in Sanfilippo syndrome. The study, “Recommendations on clinical trial design for treatment of Mucopolysaccharidosis Type III” was published in the journal Orphanet Journal of Rare Diseases. Sanfilippo syndrome, or mucopolysaccharidosis type III, belongs…

Later Stages of Sanfilippo Syndrome Type A Associated with Severe Decline in Communication, Daily Living, Socialization and Motor Skills, Study Reveals

Patients with Sanfilippo syndrome type A function below a two-year-old level in terms of communication, daily living skills and socialization, according to a small follow-up study of the later stages of the disorder. Motor skills were slightly more preserved. The study also revealed that parent burden shifts from behavioral control…

Genetic SRT May Be Promising Treatment for Sanfilippo Syndrome, Similar Disorders, Review Finds

Genetic substrate reduction therapy (gSRT) may become a promising new avenue to treat patients with lysosomal storage disorders such as Sanfilippo syndrome, according to a review study. The review, “Genetic Substrate Reduction Therapy: A Promising Approach for Lysosomal Storage Disorders,” published in Diseases, focused on gathering, summarizing, and discussing…