2025
article
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Aliya Khan, B.L. Clarke, Mishaela R. Rubin, Michael S. Ominsky et autres
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2025
article
OpenAlex
Aliya Khan, Andrea Palermo, Mishaela R. Rubin, Peter E. H. Schwarz et autres
ca, us, dk, it, de
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2025
conference-abstract
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Andrea Palermo, Aliya Khan, Mishaela R. Rubin, Peter E. H. Schwarz et autres
Accès ouvert
2025
article
OpenAlex
Aristides K Maniatis, Paul Thornton, Ulhas M. Nadgir, Elpis Vlachopapadopoulou et autres
Introduction: This international, Phase 3, open-label extension trial evaluated the long-term safety and efficacy of once-weekly lonapegsomatropin in children with growth hormone deficiency (GHD). METHODS: Conducted across 63 sites (15 countries), the enliGHten trial enrolled children with GHD who previously participated in …
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2025
article
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Norbert Albers, Sarah Cadarette, Benjamin G. Feakins, María Arregui et autres
Long-acting growth hormone (LAGH) has the potential to improve adherence and outcomes over daily somatropin in growth hormone deficiency (GHD). Whereas daily somatropin products are molecularly identical, LAGHs are molecularly distinct; additional moieties or mechanisms that prolong LAGH action confer unique pharmacodynamic/pharmacokinetic …
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2025
article
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Richard N. Channick, Sarah Medrek, Marion Delcroix, Seán Gaine et autres
BACKGROUND Age affects disease severity and patient outcomes in pulmonary arterial hypertension. This post-hoc analysis identified prognostic age groups and associated macitentan/selexipag treatment effects. METHODS Randomized trials evaluated macitentan (SERAPHIN; NCT00660179) and selexipag (GRIPHON; NCT01106014) versus placebo (primary endpoint: time to morbidity/mortality …
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2024
article
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Philippe Backeljauw, Aristides K Maniatis, M. Jennifer Abuzzahab, Steven D. Chernausek et autres
Abstract Disclosure: P.F. Backeljauw: Advisory Board Member; Self; Ascendis Pharma, Inc., Novo Nordisk, BioMarin. Consulting Fee; Self; Ascendis Pharma, Inc., Novo Nordisk, BioMarin, Cavalry, Upsher-Smith. Speaker; Self; Ascendis Pharma, Inc., BioMarin. A.K. Maniatis: Advisory Board Member; Self; Alexion Pharmaceuticals, Inc., Ascendis Pharma, …
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2024
article
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Robert P. Frantz, Shashank Desai, Gregory A. Ewald, Verónica Franco et autres
Abstract Macitentan is a dual endothelin receptor antagonist (ERA) approved for treating pulmonary arterial hypertension (PAH). SOPRANO evaluated the efficacy and safety of macitentan versus placebo in pulmonary hypertension (PH) patients after left ventricular assist device (LVAD) implantation. SOPRANO was a phase …
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2023
article
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Paul L. Hofman, Paul Thornton, Ulhas Nadgir, Paul Saenger et autres
Abstract Disclosure: P. Hofman: Speaker; Self; Novo Nordisk, Eli Lilly & Company. P.S. Thornton: Research Investigator; Self; Ascendis, Pfizer, Inc., Opko, Zealand, Rezolute, Spruce. Other; Self; Johnson &Johnson, Rezolute. U.M. Nadgir: None. P. Saenger: None. E. Chertok: None. E.M. Aghajanova: Speaker; Self; …
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2023
article
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Karim El‐Kersh, Carol Zhao, Gregory Elliott, Harrison W. Farber et autres
us
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2022
article
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Adaani Frost, Carol Zhao, Harrison W. Farber, Raymond L. Benza et autres
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2021
article
OpenAlex
Vallerie V. McLaughlin, Kelly Chin, Harrison W. Farber, Murali M. Chakinala et autres