Výsledky vyhledávání - "Tony Koutsoukos"

A randomized, double‐blind trial of triheptanoin for drug‐resistant epilepsy in glucose transporter 1 deficiency syndrome

Autor: Pasquale Striano, Stéphane Auvin, Abigail Collins, Rita Horvath, Ingrid E. Scheffer, Michal Tzadok, Ian Miller, Mary Kay Koenig, Adrian Lacy, Ronald Davis, Angela Garcia‐Cazorla, Russell P. Saneto, Melanie Brandabur, Susan Blair, Tony Koutsoukos, Darryl De Vivo

Publikováno v: Epilepsia. 63:1748-1760

This study was undertaken to evaluate efficacy and long-term safety of triheptanoin in patients1 year old, not on a ketogenic diet, with drug-resistant seizures associated with glucose transporter 1 deficiency syndrome (Glut1DS).UX007G-CL201 was a ra

Externí odkaz: https://explore.openaire.eu/search/publication?articleId=doi_dedup___::b7ee0e7b5cc09d8c2458643b7e85b89e
https://doi.org/10.1111/epi.17263

Zobrazit plný text záznamu

Retrospective chart review of urinary glycosaminoglycan excretion and long-term clinical outcomes of enzyme replacement therapy in patients with mucopolysaccharidoses

Autor: Tony Koutsoukos, Deborah Marsden, Karen Tylee, Simon Jones, Emil D. Kakkis, Sarah Phillippo, Jennifer Sniadecki

Publikováno v: Molecular Genetics and Metabolism. 130:255-261

Background Mucopolysaccharidoses (MPS) are a group of rare, inherited metabolic diseases that result from a deficiency in one of several lysosomal enzymes essential for stepwise glycosaminoglycan (GAG) degradation, leading to GAG accumulation and wid

Externí odkaz: https://explore.openaire.eu/search/publication?articleId=doi_dedup___::75a3a15979c718a71116e5bb0d385f48
https://doi.org/10.1016/j.ymgme.2020.06.004

Zobrazit plný text záznamu

Results from a 3-year Non-interventional, Observational Disease Monitoring Program in Adults with GNE Myopathy

Autor: Emil D. Kakkis, Ivailo Tournev, Camille L Bedrosian, Mark A. Tarnopolsky, Anthony Behin, Alison Skrinar, Hanns Lochmüller, Tahseen Mozaffar, Tony Koutsoukos, J. Shah, Oksana Pogoryelova, Rita Horvath

Publikováno v: Journal of neuromuscular diseases, vol 8, iss 2
Journal of Neuromuscular Diseases
Journal of Neuromuscular Diseases, 2021, 8 (2), pp.225-234. ⟨10.3233/jnd-200565⟩
Journal of Neuromuscular Diseases, IOS Press, 2021, 8 (2), pp.225-234. ⟨10.3233/jnd-200565⟩

Author(s): Lochmuller, Hanns; Behin, Anthony; Tournev, Ivailo; Tarnopolsky, Mark; Horvath, Rita; Pogoryelova, Oksana; Shah, Jinay; Koutsoukos, Tony; Skrinar, Alison; Kakkis, Emil; Bedrosian, Camille L; Mozaffar, Tahseen | Abstract: BackgroundGNE myop

Externí odkaz: https://explore.openaire.eu/search/publication?articleId=doi_dedup___::e879c36df374f48285e7a67cb79dc7c9
https://escholarship.org/uc/item/4rd9372s

Zobrazit plný text záznamu

A phase 3 randomized study evaluating sialic acid extended-release for GNE myopathy

Autor: Tony Koutsoukos, J. Shah, Alexander Lai, Catherine Woods, Emil D. Kakkis, Hank Mansbach, Alison Skrinar, Yoseph Caraco, Mark A. Tarnopolsky, Heather Lau, Ivailo Tournev, Anthony Behin, Massimiliano Mirabella, Oksana Pogoryelova, Tahseen Mozaffar, Hanns Lochmüller

Publikováno v: Neurology

Objective To investigate the efficacy and safety of aceneuramic acid extended-release (Ace-ER), a treatment intended to replace deficient sialic acid, in patients with GNE myopathy. Methods UX001-CL301 was a phase 3, double-blind, placebo-controlled,

Externí odkaz: https://explore.openaire.eu/search/publication?articleId=doi_dedup___::61cfd73147d94f0f4b5ec8afd76d6ffd
http://hdl.handle.net/10807/135646

Zobrazit plný text záznamu

Aceneuramic Acid Extended Release Administration Maintains Upper Limb Muscle Strength in a 48-week Study of Subjects with GNE Myopathy: Results from a Phase 2, Randomized, Controlled Study

Autor: Zohar Argov, Alan Pestronk, Ruhi Ahmed, Julia Martinisi, Yoseph Caraco, Emil D. Kakkis, Alison Skrinar, Heather Lau, Tony Koutsoukos, Perry B. Shieh

Publikováno v: Journal of Neuromuscular Diseases

Background GNE Myopathy (GNEM) is a progressive adult-onset myopathy likely caused by deficiency of sialic acid (SA) biosynthesis. Objective Evaluate the safety and efficacy of SA (delivered by aceneuramic acid extended-release [Ace-ER]) as treatment

Externí odkaz: https://explore.openaire.eu/search/publication?articleId=doi_dedup___::359935aed7218713aa0104f6e7acfc42
https://pubmed.ncbi.nlm.nih.gov/27854209

Zobrazit plný text záznamu

Randomized Phase 2b Study of Pralatrexate Versus Erlotinib in Patients With Stage IIIB/IV Non–Small-Cell Lung Cancer (NSCLC) After Failure of Prior Platinum-Based Therapy

Autor: Garry Alan Weems, Karen Kelly, Erzsébet Juhász, Jyoti D. Patel, Christopher G. Azzoli, Peter Y.Z. Jiang, Nicholas Iannotti, Petr Zatloukal, José Rodrigues Pereira, David Bodkin, Istvan Albert, Tony Koutsoukos

Publikováno v: Journal of Thoracic Oncology. 7:1041-1048

Introduction Pralatrexate, a folate analogue targeting dihydrofolate reductase, has antitumor activity in non–small-cell lung cancer (NSCLC). This randomized phase 2b trial was designed to further evaluate pralatrexate activity in NSCLC by estimati

Externí odkaz: https://explore.openaire.eu/search/publication?articleId=doi_dedup___::8588e5c7aa858dafbe60a22f9d43cee7
https://doi.org/10.1097/jto.0b013e31824cc66c

Zobrazit plný text záznamu

Assessment of futility in clinical trials

Autor: Qi Jiang, Steven M. Snapinn, Mon-Gy Chen, Tony Koutsoukos

Publikováno v: Pharmaceutical Statistics. 5:273-281

The term 'futility' is used to refer to the inability of a clinical trial to achieve its objectives. In particular, stopping a clinical trial when the interim results suggest that it is unlikely to achieve statistical significance can save resources

Externí odkaz: https://explore.openaire.eu/search/publication?articleId=doi_dedup___::7311486501f7a6c0bf402e4606f89b26
https://doi.org/10.1002/pst.216

Zobrazit plný text záznamu

Identification of an active, well-tolerated dose of pralatrexate in patients with relapsed or refractory cutaneous T-cell lymphoma

Autor: Andrei R. Shustov, Patricia L. Myskowski, Michael Saunders, Francine M. Foss, Tony Koutsoukos, Youn H. Kim, David C. Fisher, Steven M. Horwitz, Owen A. O'Connor, Maria Delioukina, Jasmine Zain, Mary Jo Lechowicz, Nancy L. Bartlett, Madeleine Duvic

Publikováno v: Blood. 119(18)

Systemic treatment for cutaneous T-cell lymphoma (CTCL) involves the use of less aggressive, well-tolerated therapies. Pralatrexate is a novel antifolate with high affinity for reduced folate carrier-1. A dose de-escalation strategy identified recomm

Externí odkaz: https://explore.openaire.eu/search/publication?articleId=doi_dedup___::8c0628fa2c7bc76902514d644b3f408e
https://pubmed.ncbi.nlm.nih.gov/22394596

Zobrazit plný text záznamu

Vyhledávací nástroje:

Upřesnit hledání