GM1 Gangliosidosis Pre-Clinical Publication

We believe that intracerebroventricular enzyme replacement therapy holds exciting potential to treat rare pediatric neurological diseases, including GM1 Gangliosidosis, which is in our pre-clinical development pipeline (AX 552).


Download Intracerebroventricular enzyme replacement therapy with β-galactosidase reverses brain pathologies due to GM1 gangliosidosis in mice, published in The Journal of Biological Chemistry (2020)



Related Posts

See All

Allievex obtained an exclusive worldwide license for tralesinidase alfa from BioMarin (BMN 250) in October 2019 for the treatment of patients with Sanfilippo syndrome type B. Review pre-clinical publi

Screen Shot 2021-01-25 at 4.58.05 PM.png