Clinical trials are an essential step in treatment development, yet the many people they could potentially help are either unfamiliar with the studies or unsure about what they entail. To help remedy that, the Sanfilippo Children’s Foundation has developed fact sheets about clinical trials. The family resource…
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…
Many efforts have been made to improve care and find a cure for patients with Sanfilippo disease. While some pursuits have fallen short, others hold promise. In the study “How close are we to therapies for Sanfilippo disease?” researchers at University of Gdańsk, Poland, reviewed the latest…
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