The case of two siblings with Sanfilippo syndrome type B who had the same disease-causing mutations but showed dramatically…
Articles by Marisa Horak, MS
Treatment with SOBI003, a now-discontinued experimental therapy for Sanfilippo syndrome type A, was generally well-tolerated and reduced heparan sulfate…
Levels of a protein called cystatin C are elevated in the lungs of people with Sanfilippo syndrome and related…
Sanfilippo syndrome affected the hearts of two sisters with the rare genetic disorder, without causing any of its more…
A team led by researchers at Lysogene has developed a system of four “stages” to describe the severity of…
Communication challenges and mobility difficulties are among the most impactful symptoms for children with Sanfilippo syndrome, caregivers reported in…
The Cure Sanfilippo Foundation is supporting a research project that aims to characterize fruit fly models…
A clinical trial that had been testing the experimental gene therapy ABO-102 in people with advanced syndrome type A…
A coalition of patient advocacy organizations has published a Global Roadmap for Sanfilippo Syndrome Therapies, in an effort to…
In a world-first, a team of scientists in Australia has developed a model of Sanfilippo syndrome type A in…