Effect of systemic high dose enzyme replacement therapy on the improvement of CNS defects in a mouse model of mucopolysaccharidosis type II

Conclusions:
These results suggest that high-dose ERT given to mice of earlier ages may play a role in preventing GAG accumulation and preventing CNS damage in IDS KO mice. Therefore, ERT above the present standard dose, starting in early childhood, could be a promising treatment regimen for reducing neurological impairment in Hunter syndrome.

Source: Orphanet Journal of Rare Diseases - October 31, 2015 Category: Internal Medicine Authors: Sung Yoon ChoJeehun LeeAh-Ra KoMin Jung KwakSujin KimYoung Bae SohnSung Won ParkDong-Kyu Jin Source Type: research