Results 51 to 60 of about 285 (98)

A perfectly imperfect engine: Utilizing the digital twin paradigm in pulmonary hypertension

Pulmonary Circulation, Volume 14, Issue 2, April 2024.
Abstract Pulmonary hypertension (PH) is a severe medical condition with a number of treatment options, the majority of which are introduced without consideration of the underlying mechanisms driving it within an individual and thus a lack of tailored approach to treatment.
Melody Walker, Helen Moore, Ali Ataya, Ann Pham, Paul A. Corris, Reinhard Laubenbacher, Andrew J. Bryant   +6 more
wiley   +1 more source

No Recipe for Quantitative Systems Pharmacology Model Validation, but a Balancing Act Between Risk and Cost

Clinical Pharmacology &Therapeutics, Volume 115, Issue 1, Page 25-28, January 2024.
Vincent Lemaire, Chuanpu Hu, Piet H. van der Graaf, Steve Chang, Weirong Wang   +4 more
wiley   +1 more source

P349: Plasma lyso-sphingomyelin, biomarker for acid sphingomyelinase deficiency: Correlations with baseline disease and response to olipudase alfa treatment in clinical trials

Genetics in Medicine Open, 2023
Melissa Wasserstein, Roberto Giugliani, Simon Jones, Robin Lachmann, Maurizio Scarpa, Monica Kumar, Nicole Armstrong, Mario Aguiar   +7 more
doaj   +1 more source

Olipudase alfa for the treatment of acid sphingomyelinase deficiency (ASMD): safety and efficacy in adults treated for 30months [PDF]

Molecular Genetics and Metabolism, 2017
Ana Cristina Puga, Melissa Wasserstein, George Diaz, Robin Lachmann, Marie-Helene Jouvin, Indrani Nandy, Allena Ji, Haig Ingulizian   +7 more
openaire   +1 more source

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

Genet Med, 2021
Diaz GA, Jones SA, Scarpa M, Mengel KE, Giugliani R, Guffon N, Batsu I, Fraser PA, Li J, Zhang Q, Ortemann-Renon C.   +10 more
europepmc   +1 more source

O33: Impact of p.Arg610del neuroprotective genotype on disease burden and treatment response in adults with acid-sphingomyelinase deficiency: ASCEND olipudase alfa trial

Genetics in Medicine Open
Melissa Wasserstein, Monica Kumar, Nicole Armstrong, Sefika Uslu Cil, Arya Firoozan, Lisa Underhill, Robin Lachmann   +6 more
doaj   +1 more source

P206: Continued clinical improvements in adults with acid sphingomyelinase deficiency after 3-5 Years of olipudase alfa treatment: Final ASCEND trial results

Genetics in Medicine Open
Melissa Wasserstein, Renata Gallagher, Antonio Barbato, Roberto Giugliani, Norberto Guelbert, Julia Hennermann, Takayuki Ikezoe, Robin Lachmann, Olivier Lidove, Paulina Mabe, Eugen Mengel, Maurizio Scarpa, Ebubekir Senates, Michel Tchan, Jesus Villarrubia, Beth Thurberg, Nicole Armstrong, Shruti Srivastava, Urszula Wisniewska, Monica Kumar   +19 more
doaj   +1 more source

P034: A real-world evidence study into the usage of olipudase alfa for acid sphingomyelinase deficiency in the United States

Genetics in Medicine Open
Judy Hull, Neeraj Rawat, Laurence Pollissard, Gavin Harper, Joe Conyers, Rabiyah Sahar   +5 more
openaire   +1 more source

Screening for acid sphingomyelinase deficiency in patients with an interstitial lung disease. [PDF]

Orphanet J Rare Dis
Vermant M, Van Raemdonck I, Veyt N, Carlon MS, Cortesi EE, De Crem N, De Rooy M, Follet T, Gogaert S, Vanaudenaerde B, Cassiman D, De Sadeleer LJ, Wuyts WA.   +12 more
europepmc   +1 more source

Coexistence of acid sphingomyelinase deficiency type A/B and Arnold-Chiari malformation: a novel case report. [PDF]

Front Pediatr
Kamalova A, Rakhmaeva R, Sageeva G, Safiullina R, Raimova A, Gaichik E, Nasr D, Gobarah AA, Arafat A.   +8 more
europepmc   +1 more source