|
Initiation of long-term ERT with cerliponase alfa at 300 mg (or age-appropriate) dose every other week through intraventricular infusion is suggested in non-classical TPP1 deficiency patients after confirmed diagnosis and agreement between parents and provider, as long as no contraindications to therapy exist. Initiation of long-term ERT with cerliponase alfa at 300 mg (or age-appropriate) dose every other week through intraventricular infusion is recommended in classical CLN2 patients with the potential to benefit from this therapy
|
37
|
C
|
84
|
|
Disease-modifying treatment with a licensed therapy ideally should be delivered by a team experienced in the management of CLN2 disease and use of any required devices. For current ERT treatment for CLN2 disease, this includes brain intraventricular devices
|
39
|
C
|
93
|
|
There is no evidence currently that HSCT benefits patients with CLN2 and at this time is not recommended or approved as a treatment
|
34
|
C
|
93
|
|
Intraventricular devices should be placed under general anaesthesia by a very experienced paediatric neurosurgeon
|
36
|
C
|
92
|
|
Intraventricular device should only be accessed by a trained individual to limit/ minimise complications
|
39
|
C
|
95
|
