Author Archives: Marisa Wexler MS

Sanfilippo B Enzyme Replacement Therapy M041 Can Get Into Cells

M041, an enzyme replacement therapy M6P Therapeutics (M6PT) is developing for Sanfilippo syndrome type B, can effectively be transported into cells, proof-of-concept preclinical data show. With data showing such “increased efficiency … M041 is a potential novel recombinant enzyme therapy for Sanfilippo B syndrome,” M6PT said in a…

2 New NAGLU Mutations Possibly Linked to Sanfilippo Type B

Two never-before-reported mutations were found in Sanfilippo syndrome type B patients, with one likely to be disease-causing, a recent study reported. How the other identified mutation relates to this disease is not yet known, its researchers said. The study, “Clinical and genetic features of 13 patients with…

EMDR May Be Effective for Treating Parents’ PTSD

Eye Movement Desensitization and Reprocessing (EMDR) can help to ease symptoms of post-traumatic stress disorder (PTSD) in parents of children with Sanfilippo syndrome, a case study suggests. The study, “Reducing posttraumatic stress in parents of patients with a rare inherited metabolic disorder using eye movement desensitization…

#AANAM – Gene Therapy Shows Promise in Early Type A Disease

Treatment with ABO-102, an investigational gene therapy being developed by Abeona Therapeutics, normalized development in children with Sanfilippo syndrome type A, according to new data from a clinical trial. The data indicate that the gene therapy is especially effective when given early in life, before the disease has…