https://pubmed.ncbi.nlm.nih.gov/41737901/
Intrathecal idursulfase-IT in children younger than 3 years with neuronopathic...
Data from a phase 2/3, randomized, controlled, open-label, multicenter trial in children with neuronopathic mucopolysaccharidosis II (MPS II; Hunter syndrome)...
in childrenintrathecal
https://pubmed.ncbi.nlm.nih.gov/18509745/
Management of non-neuronopathic Gaucher disease with special reference to pregnancy, splenectomy,...
Enzyme replacement was introduced as treatment for non-neuronopathic Gaucher disease more than 15 years ago. To ensure the best use of this costly ultra-orphan...
gaucher disease