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ALLMedicine™ Acid Sphingomyelinase Deficiency Center

Research & Reviews  27 results

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

Sep 27th, 2021 - The maximum study duration per patient is 9 years or until marketing approval whichever comes first, unless the patient decides to enter another olipudase alfa clinical trial within the 9-year period prior to marketing approval. If marketing appro...

Prospective study of the natural history of chronic acid sphingomyelinase deficiency in...
https://www.ncbi.nlm.nih.gov/pmc/articles/PMC8111900
Orphanet Journal of Rare Diseases; McGovern MM, Wasserstein MP et. al.

May 12th, 2021 - Acid sphingomyelinase deficiency (ASMD) (also known as Niemann-Pick disease types A and B) is a rare and debilitating lysosomal storage disorder. This prospective, multi-center, multinational longitudinal study aimed to characterize the clinical f...

Interstitial lung disease in lysosomal storage disorders.
https://doi.org/10.1183/16000617.0363-2020
European Respiratory Review : an Official Journal of the ... Borie R, Crestani B et. al.

May 1st, 2021 - Lysosomes are intracellular organelles that are responsible for degrading and recycling macromolecules. Lysosomal storage diseases (LSDs) are a group of inherited diseases caused by mutations affecting genes that encode the function of the lysosom...

One-year results of a clinical trial of olipudase alfa enzyme replacement therapy in pe...
https://www.ncbi.nlm.nih.gov/pmc/articles/PMC8354848
Genetics in Medicine : Official Journal of the American C... Diaz GA, Jones SA et. al.

Apr 21st, 2021 - To assess olipudase alfa enzyme replacement therapy for non-central nervous system manifestations of acid sphingomyelinase deficiency (ASMD) in children. This phase 1/2, international, multicenter, open-label trial (ASCEND-Peds/NCT02292654) admini...

Altered Macrophage Function Associated with Crystalline Lung Inflammation in Acid Sphin...
https://www.ncbi.nlm.nih.gov/pmc/articles/PMC8086042
American Journal of Respiratory Cell and Molecular Biology; Poczobutt JM, Mikosz AM et. al.

Mar 5th, 2021 - Deficiency of ASM (acid sphingomyelinase) causes the lysosomal storage Niemann-Pick disease (NPD). Patients with NPD type B may develop progressive interstitial lung disease with frequent respiratory infections. Although several investigations usi...

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Clinicaltrials.gov  2 results

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

Sep 27th, 2021 - The maximum study duration per patient is 9 years or until marketing approval whichever comes first, unless the patient decides to enter another olipudase alfa clinical trial within the 9-year period prior to marketing approval. If marketing appro...

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

Feb 1st, 2021 - 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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