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)
