Any child who experiences developmental delay or intellectual disability by age 18 should undergo genetic sequencing as a first- or…
Articles by Marisa Horak, MS
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…
Taysha Gene Therapies and Invitae have announced a partnership to support Invitae’s Detect Lysosomal Storage Diseases (Detect…
A majority of rare disease patients using telehealth during the COVID-19 pandemic thought the experience positive, and many would like…
Problems with one or more senses are common in people with Sanfilippo syndrome and other mucopolysaccharidoses (MPSs), but…
Seelos Therapeutics announced it has received a positive opinion on an orphan drug designation for SLS-005 (trehalose)…