The Foundation recently developed Sanfilippo clinical trial factsheets for families, which are available on our website here. Factsheets are organised by subtype, providing information on what clinical trials are available, some eligibility criteria and current reported progress.
While there are no approved therapies currently available for patients with Sanfilippo, recent years have seen an increase in the number of Sanfilippo clinical trials. Now, there are nine trials currently taking place around the world (five are recruiting), all at various stages, involving different Sanfilippo subtypes and kinds of therapies.
The factsheets were prepared with the help of a $10,000 Community Grant from CommBank, awarded to the Sanfilippo Children’s Foundation to provide up-to-date material on emerging therapies for families affected by Sanfilippo Syndrome. This grant will also go towards the employment of the Foundation’s Research Program Coordinator, Danielle Cini.
Clinical trials are an essential step in therapy development, designed to test whether a potential therapy is safe and effective in humans. Clinical trials build on years of work in the lab, designing, developing and testing a therapy. A well-executed clinical trial that proves a therapy to be safe and effective offers the best chance at getting regulatory approval for widespread use in patients with Sanfilippo.
These are certainly exciting times in Sanfilippo research, and we will keep everyone updated on clinical trial progress as it unfolds.