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About 123 results

ALLMedicine™ Acid Sphingomyelinase Deficiency Center

Research & Reviews  40 results

Data Analysis of Adult and Pediatric Participants With Acid Sphingomyelinase Deficiency (ASMD) on Early Access to Olipudase Alfa in France
https://clinicaltrials.gov/ct2/show/NCT05359276

Jun 14th, 2022 - Approximate duration of enrollment: 18 months Total study duration: approximately 3 years This is a national, multicenter observational retrospective and prospective cohort data collection study. Retrospective is defined as collection of data from...

A Long-Term Study of Olipudase Alfa in Patients With Acid Sphingomyelinase Deficiency
https://clinicaltrials.gov/ct2/show/NCT02004704

Jun 6th, 2022 - The maximum study duration per patient is 9 years or until olipudase alfa becomes commercially accessible (see maximum duration below), whichever comes first, unless the patient decides to enter another olipudase alfa clinical trial within the 9-y...

Olipudase Alfa: First Approval.
https://doi.org/10.1007/s40265-022-01727-x
Drugs Keam SJ

Jun 1st, 2022 - Olipudase alfa (XENPOZYME®) is a recombinant human acid sphingomyelinase that has been developed by Sanofi, for the treatment of acid sphingomyelinase deficiency (ASMD). Olipudase alfa catalyses the hydrolysis of sphingomyelin accumulated in hepat...

A 2-bp deletion mutation in SMPD1 gene leading to lysosomal acid sphingomyelinase defic...
https://doi.org/10.1515/jpem-2021-0480
Journal of Pediatric Endocrinology & Metabolism : JPEM; Kang H, Zhou M et. al.

May 27th, 2022 - Niemann-Pick disease type A (NPDA, MIM: 257200) is an autosomal recessive sphingolipidosis caused by lysosomal acid sphingomyelinase (ASM) deficiency. A cluster of genes located at chromosome 11p15 have been reported to be imprinted genes, such as...

Efficacy, Safety, Pharmacodynamic, and Pharmacokinetics Study of Olipudase Alfa in Patients With Acid Sphingomyelinase Deficiency
https://clinicaltrials.gov/ct2/show/NCT02004691

May 24th, 2022 - The total duration per participant will be at least 3 years and up to 5 years and 3 months. This includes up to approximately two month of screening, 52 weeks of primary analysis period, up to 4 years and 3 months of extension treatment period, an...

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Clinicaltrials.gov  8 results

Data Analysis of Adult and Pediatric Participants With Acid Sphingomyelinase Deficiency (ASMD) on Early Access to Olipudase Alfa in France
https://clinicaltrials.gov/ct2/show/NCT05359276

Jun 14th, 2022 - Approximate duration of enrollment: 18 months Total study duration: approximately 3 years This is a national, multicenter observational retrospective and prospective cohort data collection study. Retrospective is defined as collection of data from...

A Long-Term Study of Olipudase Alfa in Patients With Acid Sphingomyelinase Deficiency
https://clinicaltrials.gov/ct2/show/NCT02004704

Jun 6th, 2022 - The maximum study duration per patient is 9 years or until olipudase alfa becomes commercially accessible (see maximum duration below), whichever comes first, unless the patient decides to enter another olipudase alfa clinical trial within the 9-y...

Efficacy, Safety, Pharmacodynamic, and Pharmacokinetics Study of Olipudase Alfa in Patients With Acid Sphingomyelinase Deficiency
https://clinicaltrials.gov/ct2/show/NCT02004691

May 24th, 2022 - The total duration per participant will be at least 3 years and up to 5 years and 3 months. This includes up to approximately two month of screening, 52 weeks of primary analysis period, up to 4 years and 3 months of extension treatment period, an...

Safety, Tolerability, PK, and Efficacy Evaluation of Repeat Ascending Doses of Olipudase Alfa in Pediatric Patients <18 Years of Age With Acid Sphingomyelinase Deficiency
https://clinicaltrials.gov/ct2/show/NCT02292654

Mar 23rd, 2022 - The maximum study duration per participant was approximately 18 months (screening period: up to 60 days; treatment period: 64 weeks; post-treatment period: up to 37 days, not applicable if participants enrolled in a long-term extension treatment t...

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News  1 results

FDA grants breakthrough therapy designation to Genzyme's Olipudase Alfa
https://www.reuters.com/article/us-sanofi-fda/fda-grants-breakthrough-therapy-designation-to-genzymes-olipudase-alfa-idUSKBN0OK0CT20150604

Jun 4th, 2015 - PARIS (Reuters) - Sanofi and its subsidiary Genzyme said on Thursday that the U.S. Food and Drug Administration (FDA) has granted breakthrough therapy designation to olipudase alfa. This enzyme replacement therapy is being investigated for the tre...

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