One of the children participating in the Phase 2/3 trial of LYS-SAF302, Lysogene’s gene therapy candidate for…
Articles by Joana Carvalho, PhD
An accurate and early diagnosis of Sanfilippo syndrome type CÂ can be difficult, as this subtype is quite rare, but…
Physicians should be aware that milder and slow-progressing forms of Sanfilippo syndrome may not be associated with severe cognitive…
After a pivotal start in 2019 to its clinical program for Sanfilippo syndrome type A, Lysogene has announced…
A multidisciplinary approach tackling not only the behaviors and symptoms of children with Sanfilippo type B, but also the…
Scientists have developed a new method to gather samples of cerebrospinal fluid (CSF) — the liquid that circulates in the…
Using different cellular models of Sanfilippo syndrome type B, researchers have found that exposure time and dose significantly…
Exome sequencing data — the DNA sequence of all genes encoding for proteins — that include the contribution of…
Restoring the activity of the alpha-N-acetylglucosaminidase (NAGLU) enzyme using enzyme replacement therapy (ERT) is well-tolerated by patients with…
Combined behavioral and pharmacological therapy seems to be the best course of action in treating behavioral and sleep problems in…