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Unexpected Gaucher disease in a case of steroid-resistant nephrotic syndrome. [PDF]

open access: yesPediatr Nephrol
Gehad MH   +4 more
europepmc   +1 more source

Sinbaglustat is efficacious in GM2 gangliosidosis primarily through inhibition of GBA2 rather than GCS. [PDF]

open access: yesMol Ther Adv
Steiner MA   +8 more
europepmc   +1 more source

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