Safety and efficacy of rodatristat ethyl for the treatment of pulmonary arterial hypertension (ELEVATE-2): a dose-ranging, randomised, multicentre, phase 2b trial.

Autor: Sitbon O; Faculty of Medicine, Université Paris-Saclay, Le Kremlin-Bicêtre, France; INSERM UMR_S 999 (HPPIT), Le Kremlin-Bicêtre, France; Department of Respiratory and Intensive Care Medicine, Assistance Publique Hôpitaux de Paris, Hôpital Bicêtre, ERN-LUNG, Le Kremlin-Bicêtre, France., Skride A; Rare Diseases Unit VSIA Pauls Stradins Clinical University Hospital, Riga Stradiņš University, Riga, Latvia., Feldman J; Summit Health, Bend, OR, USA., Sahay S; Houston Methodist Lung Center, Houston Methodist Hospital, Houston, TX, USA., Shlobin OA; Advanced Lung Disease and Transplant Program, Inova Fairfax Hospital, Falls Church, VA, USA., McLaughlin V; Division of Cardiovascular Medicine, Department of Internal Medicine, University of Michigan Health System, Ann Arbor, MI, USA., Ghofrani HA; Department of Pneumology, University of Giessen and Marburg, Giessen, Germany., Langleben D; Center for Pulmonary Vascular Disease, Division of Cardiology, Jewish General Hospital, McGill University, Montreal, QC, Canada., Parsley E; Bellerophon Therapeutics, Warren, NJ, USA., D'Souza G; GDS ClinicalZest, Dover, UK., Marmon T; Marmon Biostatistics, Seattle, WA, USA., Kamau-Kelley W; Sumitomo Pharma America (formerly Enzyvant), Morrisville, NC, USA., Jones R; Sumitomo Pharma America (formerly Enzyvant), Morrisville, NC, USA., Grewal R; Sumitomo Pharma America (formerly Enzyvant), Morrisville, NC, USA., Wring S; Sumitomo Pharma America (formerly Enzyvant), Morrisville, NC, USA., Palacios M; Sumitomo Pharma America (formerly Enzyvant), Morrisville, NC, USA., Naik H; Sumitovant Biopharma, Boston, MA, USA., Denning J; Sumitomo Pharma America (formerly Enzyvant), Morrisville, NC, USA., Lazarus HM; Sumitomo Pharma America (formerly Enzyvant), Morrisville, NC, USA., Humbert M; Faculty of Medicine, Université Paris-Saclay, Le Kremlin-Bicêtre, France; INSERM UMR_S 999 (HPPIT), Le Kremlin-Bicêtre, France; Department of Respiratory and Intensive Care Medicine, Assistance Publique Hôpitaux de Paris, Hôpital Bicêtre, ERN-LUNG, Le Kremlin-Bicêtre, France. Electronic address: marc.humbert@aphp.fr.

Publikováno v: The Lancet. Respiratory medicine [Lancet Respir Med] 2024 Nov; Vol. 12 (11), pp. 865-876. Date of Electronic Publication: 2024 Sep 19.

ACROBAT Edge: Safety and Efficacy of Switching Injected SRLs to Oral Paltusotine in Patients With Acromegaly.

Autor: Gadelha MR; Neuroendocrinology Research Center/Endocrinology Division, Medical School and Hospital Universitario Clementino Fraga Filho, Universidade Federal do Rio de Janeiro, Rio de Janeiro, CEP 21941-913, Brazil., Gordon MB; Allegheny Neuroendocrinology Center, Division of Endocrinology, Allegheny General Hospital, Pittsburgh, Pennsylvania 15212, USA., Doknic M; Neuroendocrine Department, Clinic for Endocrinology, Diabetes and Metabolic Diseases, University Clinical Center of Serbia; Faculty of Medicine, University of Belgrade, Belgrade 11000, Serbia., Mezősi E; First Department of Internal Medicine, Faculty of Medicine, University of Pécs Medical School, Pécs 7624, Hungary., Tóth M; Department of Internal Medicine and Oncology, Faculty of Medicine, Semmelweis University, Budapest 1085, Hungary., Randeva H; Warwickshire Institute for the Study of Diabetes, Endocrinology and Metabolism (WISDEM), University Hospitals Coventry and Warwickshire NHS Trust, Coventry CV2 2DX, UK.; Division of Biomedicine, Warwick Medical School, University of Warwick, Coventry CV4 7AL, UK., Marmon T; Crinetics Pharmaceuticals Inc, San Diego, California 92121, USA., Jochelson T; Crinetics Pharmaceuticals Inc, San Diego, California 92121, USA., Luo R; Crinetics Pharmaceuticals Inc, San Diego, California 92121, USA., Monahan M; Crinetics Pharmaceuticals Inc, San Diego, California 92121, USA., Madan A; Crinetics Pharmaceuticals Inc, San Diego, California 92121, USA., Ferrara-Cook C; Crinetics Pharmaceuticals Inc, San Diego, California 92121, USA., Struthers RS; Crinetics Pharmaceuticals Inc, San Diego, California 92121, USA., Krasner A; Crinetics Pharmaceuticals Inc, San Diego, California 92121, USA.

Publikováno v: The Journal of clinical endocrinology and metabolism [J Clin Endocrinol Metab] 2023 Apr 13; Vol. 108 (5), pp. e148-e159.

Development and evaluation of the Acromegaly Symptom Diary.

Autor: Martin S; Patient-Centered Outcomes Assessment, RTI Health Solutions, Ann Arbor, MI, USA. smartin@rti.org.; Patient-Centered Outcomes Assessment, RTI Health Solutions, 200 Park Offices Drive, Research Triangle Park, NC, 27709, USA. smartin@rti.org., Bender RH; Patient-Centered Outcomes Assessment, RTI Health Solutions, 200 Park Offices Drive, Research Triangle Park, NC, 27709, USA., Krasner A; Crinetics Pharmaceuticals, Inc., San Diego, CA, USA., Marmon T; Marmon Biostatistics, Fair Oaks, CA, USA., Monahan M; Crinetics Pharmaceuticals, Inc., San Diego, CA, USA., Nelson L; Patient-Centered Outcomes Assessment, RTI Health Solutions, 200 Park Offices Drive, Research Triangle Park, NC, 27709, USA.

Publikováno v: Journal of patient-reported outcomes [J Patient Rep Outcomes] 2023 Feb 15; Vol. 7 (1), pp. 15. Date of Electronic Publication: 2023 Feb 15.

The External Validation of GLOBE and UK-PBC Risk Scores for Predicting Ursodeoxycholic Acid Treatment Response in a Large U.S. Cohort of Primary Biliary Cholangitis Patients.

Autor: Marenco-Flores, Ana¹ (AUTHOR) amarenco@bidmc.harvard.edu, Rojas Amaris, Natalia¹ (AUTHOR) nrojasam@bidmc.harvard.edu, Kahan, Tamara¹ (AUTHOR) vpatward@bidmc.harvard.edu, Sierra, Leandro¹ (AUTHOR), Barba Bernal, Romelia² (AUTHOR), Medina-Morales, Esli³ (AUTHOR), Goyes, Daniela⁴ (AUTHOR), Patwardhan, Vilas¹ (AUTHOR), Bonder, Alan¹ (AUTHOR) abonder@bidmc.harvard.edu

Publikováno v: Journal of Clinical Medicine. Aug2024, Vol. 13 Issue 15, p4497. 12p.

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