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niemann-pick diseases/maumivu ya kichwa

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NakalaMajaribio ya klinikiHati miliki
1 matokeo

Successful within-patient dose escalation of olipudase alfa in acid sphingomyelinase deficiency.

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Ingia / Ingia
BACKGROUND Olipudase alfa, a recombinant human acid sphingomyelinase (rhASM), is an investigational enzyme replacement therapy (ERT) for patients with ASM deficiency [ASMD; Niemann-Pick Disease (NPD) A and B]. This open-label phase 1b study assessed the safety and tolerability of olipudase alfa
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