Cargando…

Olipudase alfa enzyme replacement therapy for acid sphingomyelinase deficiency (ASMD): sustained improvements in clinical outcomes after 6.5 years of treatment in adults

BACKGROUND: Enzyme replacement therapy with olipudase alfa, a recombinant human acid sphingomyelinase (rhASM), is indicated for non-central nervous system manifestations of acid sphingomyelinase deficiency (ASMD) in children and adults. An ongoing, open-label, long-term study (NCT02004704) assessed...

Descripción completa

Detalles Bibliográficos
Autores principales:	Lachmann, Robin H., Diaz, George A., Wasserstein, Melissa P., Armstrong, Nicole M., Yarramaneni, Abhimanyu, Kim, Yong, Kumar, Monica
Formato:	Online Artículo Texto
Lenguaje:	English
Publicado:	BioMed Central 2023
Materias:	Research
Acceso en línea:	https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10131350/ https://www.ncbi.nlm.nih.gov/pubmed/37098529 http://dx.doi.org/10.1186/s13023-023-02700-x

Ejemplares similares

Olipudase alfa for treatment of acid sphingomyelinase deficiency (ASMD): safety and efficacy in adults treated for 30 months
por: Wasserstein, Melissa P., et al.
Publicado: (2018)

P1483: LONG-TERM IMPACT OF OLIPUDASE ALFA ENZYME REPLACEMENT THERAPY ON SPLEEN VOLUME AND HEMATOLOGIC MANIFESTATIONS IN CHILDREN AND ADULTS WITH CHRONIC ACID SPHINGOMYELINASE DEFICIENCY
por: Villarrubia, Jesus, et al.
Publicado: (2023)

Long-term safety and clinical outcomes of olipudase alfa enzyme replacement therapy in pediatric patients with acid sphingomyelinase deficiency: two-year results
por: Diaz, George A., et al.
Publicado: (2022)

Correction : Long-term safety and clinical outcomes of olipudase alfa enzyme replacement therapy in pediatric patients with acid sphingomyelinase deficiency: two-year results
por: Diaz, George A., et al.
Publicado: (2023)

PB2314: OLIPUDASE ALFA FOR ADULTS WITH ACID SPHINGOMYELINASE DEFICIENCY: IMPROVEMENTS IN CROSSOVER PLACEBO PATIENTS AND FURTHER IMPROVEMENTS IN ORIGINAL OLIPUDASE ALFA PATIENTS AFTER 2 YEARS IN ASCEND TRIAL
por: Villarrubia, J., et al.
Publicado: (2022)

Continued improvement in disease manifestations of acid sphingomyelinase deficiency for adults with up to 2 years of olipudase alfa treatment: open-label extension of the ASCEND trial
por: Wasserstein, Melissa P., et al.
Publicado: (2023)

Clearance of Hepatic Sphingomyelin by Olipudase Alfa Is Associated With Improvement in Lipid Profiles in Acid Sphingomyelinase Deficiency
por: Thurberg, Beth L., et al.
Publicado: (2016)

Recommendations for clinical monitoring of patients with acid sphingomyelinase deficiency (ASMD)
por: Wasserstein, Melissa, et al.
Publicado: (2018)

One-year results of a clinical trial of olipudase alfa enzyme replacement therapy in pediatric patients with acid sphingomyelinase deficiency
por: Diaz, George A., et al.
Publicado: (2021)

One-year results of a clinical trial of olipudase alfa enzyme replacement therapy in pediatric patients with acid sphingomyelinase deficiency
por: Diaz, George A., et al.
Publicado: (2022)

Quantitative Systems Pharmacology Modeling of Acid Sphingomyelinase Deficiency and the Enzyme Replacement Therapy Olipudase Alfa Is an Innovative Tool for Linking Pathophysiology and Pharmacology
por: Kaddi, Chanchala D., et al.
Publicado: (2018)

Olipudase Alfa in Non-CNS Manifestations of Acid Sphingomyelinase Deficiency: A Profile of Its Use
por: Syed, Yahiya Y.
Publicado: (2023)

Correction to: Olipudase Alfa in Non-CNS Manifestations of Acid Sphingomyelinase Deficiency: A Profile of Its Use
por: Syed, Yahiya Y.
Publicado: (2023)

Disease manifestations and burden of illness in patients with acid sphingomyelinase deficiency (ASMD)
por: McGovern, Margaret M., et al.
Publicado: (2017)

Changes in PCSK 9 and apolipoprotein B100 in Niemann–Pick disease after enzyme replacement therapy with olipudase alfa
por: Garside, Bethanie, et al.
Publicado: (2021)

PB2361: PREDIGA, EDUCATIONAL AND DIAGNOSIS PROJECT IN ACID SPHINGOMYELINASE DEFICIENCY DISEASE (ASMD) AND GAUCHER DISEASE (GD): RESULTS TO THE OBJECTIVE ACHIEVED OF 200 PATIENTS ANALYZED.
por: Villarrubia, J., et al.
Publicado: (2022)

Plasma lyso-sphingomyelin levels are positively associated with clinical severity in acid sphingomyelinase deficiency
por: Breilyn, Margo Sheck, et al.
Publicado: (2021)

Consensus recommendation for a diagnostic guideline for acid sphingomyelinase deficiency
por: McGovern, Margaret M., et al.
Publicado: (2017)

Benchmarking Adaptive Steered Molecular Dynamics (ASMD) on CHARMM Force Fields
por: Allen, Caley, et al.
Publicado: (2022)

Enzyme replacement therapy for children with acid sphingomyelinase deficiency in the real world: A single center experience in Taiwan
por: Pan, Yu-Wen, et al.
Publicado: (2023)

Prospective study of the natural history of chronic acid sphingomyelinase deficiency in children and adults: eleven years of observation
por: McGovern, Margaret M., et al.
Publicado: (2021)

Prevalence of Cancer in Acid Sphingomyelinase Deficiency
por: Mauhin, Wladimir, et al.
Publicado: (2021)

Novel breakthrough in the treatment of sphingomyelinase deficiency
por: Sultan, Wania, et al.
Publicado: (2023)

Improved Enzyme Replacement Therapy with Cipaglucosidase Alfa/Miglustat in Infantile Pompe Disease
por: Fiege, Lina, et al.
Publicado: (2023)

Apoptosis and signalling in acid sphingomyelinase deficient cells
por: Sillence, Dan J
Publicado: (2001)

Acid Sphingomyelinase Deficiency Ameliorates Farber Disease
por: Beckmann, Nadine, et al.
Publicado: (2019)

Autopsy pathology of infantile neurovisceral ASMD (Niemann-Pick Disease type A): Clinicopathologic correlations of a case report
por: Thurberg, Beth L.
Publicado: (2020)

An open-label clinical trial of agalsidase alfa enzyme replacement therapy in children with Fabry disease who are naïve to enzyme replacement therapy
por: Goker-Alpan, Ozlem, et al.
Publicado: (2016)

Cost-effectiveness of enzyme replacement therapy with alglucosidase alfa in adult patients with Pompe disease
por: Kanters, Tim A., et al.
Publicado: (2017)

Consensus clinical management guidelines for acid sphingomyelinase deficiency (Niemann–Pick disease types A, B and A/B)
por: Geberhiwot, Tarekegn, et al.
Publicado: (2023)

Acid Sphingomyelinase Deficiency: A Clinical and Immunological Perspective
por: Pinto, Carolina, et al.
Publicado: (2021)

Digestive enzymes and sphingomyelinase D in spiders without venom (Uloboridae)
por: Valladão, Rodrigo, et al.
Publicado: (2023)

Impact and burden of acid sphingomyelinase deficiency from a patient and caregiver perspective
por: Pokrzywinski, Robin, et al.
Publicado: (2021)

Cost-effectiveness of enzyme replacement therapy with alglucosidase alfa in classic-infantile patients with Pompe disease
por: Kanters, Tim A, et al.
Publicado: (2014)

Evaluation of the efficacy and safety of three dosing regimens of agalsidase alfa enzyme replacement therapy was underpowered
por: Ortiz, Alberto, et al.
Publicado: (2015)

Pharmacokinetic and Pharmacodynamic Evaluation of Elosulfase Alfa, an Enzyme Replacement Therapy in Patients with Morquio A Syndrome
por: Qi, Yulan, et al.
Publicado: (2014)

Odontohypophosphatasia treated with asfotase alfa enzyme replacement therapy in a toddler: a case report
por: Takagi, Mizuki, et al.
Publicado: (2020)

Quantification of lysosphingomyelin and lysosphingomyelin-509 for the screening of acid sphingomyelinase deficiency
por: Kubaski, Francyne, et al.
Publicado: (2022)

Effectiveness of agalsidase alfa enzyme replacement in Fabry disease: cardiac outcomes after 10 years’ treatment
por: Kampmann, Christoph, et al.
Publicado: (2015)

Long-term outcomes with agalsidase alfa enzyme replacement therapy: Analysis using deconstructed composite events
por: Beck, Michael, et al.
Publicado: (2017)

Cannot write session to /tmp/vufind_sessions/sess_1nkn1jm504oce9c11m2teo5bpd