Rare Diseases

Current bibliography for experts

"Olipudase Alfa" search results...

ASMD

A randomized, placebo-controlled clinical trial evaluating olipudase alfa enzyme replacement therapy for chronic ASMD in adults: Oneyear results

2022-04-26

In individuals with acid sphingomyelinase deficiency (ASMD), olipudase alfa was well tolerated and associated with meaningful and wide-ranging improvements in disease pathology and clinically relevant endpoints compared to placebo.

ASMD

One-year results of a clinical trial of olipudase alfa enzyme replacement therapy in pediatric patients with acid sphingomyelinase deficiency

2021-08-23

In this study in children with chronic ASMD, olipudase alfa was generally well-tolerated with significant, comprehensive improvements in disease pathology across a range of clinically relevant endpoints.

GAUCHER

Interstitial lung disease in lysosomal storage disorders

2021-04-29

Early diagnosis for lysosomal storage diseases (LSDs) is crucial to prevent irreversible organ damage. Early initiation of enzyme replacement therapy (ERT) can, at least in part, prevent organ failure.

NIEMANN-PICK

Changes in PCSK 9 and apolipoprotein B100 in Niemann-Pick disease after enzyme replacement therapy with olipudase alfa

2021-02-27

In this study, in which Niemann pick patients were included, it was aimed to assess the effect of enzyme replacement therapy (ERT) on lipid parameters and inflammatory markers.

NIEMANN-PICK

Long-term efficacy of olipudase alfa in adults with acid sphingomyelinase deficiency (ASMD)

2020-09-01

This is a 26-week phase 1b trial of enzyme replacement therapy (ERT) with olipudase alfa, which was followed by a long-term extension study. 5 adult patients with chronic visceral ASMD were enrolled in this trial. The findings show that hepatic clearance of sphingomyelin during olipudase alfa treatment for 42 months is associated with overall lipid profile improvements in ASMD patients.

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