Ain NU, Saith A, Ruan A, Yang R, Burton A, Mistry PK.

Eliglustat substrate reduction therapy in children with Gaucher disease type 1.

Front Pediatr. 2025 ;13:1543136. 

Ir

Weinreb NJ, Goker-Alpan O, Kishnani PS, Longo N, Burrow TA, Bernat JA, Gupta P, Henderson N, Pedro H, Prada CE, Vats D, Pathak RR, Wright E, Ficicioglu C.

The diagnosis and management of Gaucher disease in pediatric patients: Where do we go from here?

Mol Genet Metab. 2022 ;136:4-21. 

Ir