A clinical trial that had been testing the experimental gene therapy ABO-102 in people with advanced syndrome type A…
Articles by Marisa Wexler, MS
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…
Music therapy may be beneficial for children with Sanfilippo syndrome, a small study in Spain illustrates. The study, “…
Any child who experiences developmental delay or intellectual disability by age 18 should undergo genetic sequencing as a first- or…
A clinical trial extension study involving 12 children with Sanfilippo syndrome type A was terminated early, after efficacy analyses found…
M041, an enzyme replacement therapy M6P Therapeutics (M6PT) is developing for Sanfilippo syndrome type B, can effectively be…
Two never-before-reported mutations were found in Sanfilippo syndrome type B patients, with one likely to be disease-causing, a recent study…
Eye Movement Desensitization and Reprocessing (EMDR) can help to ease symptoms of post-traumatic stress disorder (PTSD) in parents of children…
Treatment with ABO-102, an investigational gene therapy being developed by Abeona Therapeutics, normalized development in children with Sanfilippo…