المصدر: A Single-Arm, Open-Label, Safety Study in Patients With Generalized Lipodystrophy Switching From Metreleptin to Mibavademab, A Leptin Receptor Agonist Antibody

Other URLs: https://vivli.org/

المصدر: Open-label, Phase 3b Study to Evaluate Effectiveness, Safety and Pharmacokinetic Parameters of Metreleptin in Patients Under 6 Years of Age With Generalised Lipodystrophy and Associated Diabetes Mellitus and/or Hypertriglyceridaemia

المصدر: Compassionate Use of Metreleptin in Previously-Treated Patients With Generalized Lipodystrophy
Malandrino N, Reynolds JC, Brychta RJ, Chen KY, Auh S, Gharib AM, Startzell M, Cochran EK, Brown RJ. Visceral fat does not contribute to metabolic disease in lipodystrophy. Obes Sci Pract. 2019 Jan 24;5(1):75-82. doi: 10.1002/osp4.319. eCollection 2019 Feb.
Meral R, Ryan BJ, Malandrino N, Jalal A, Neidert AH, Muniyappa R, Akinci B, Horowitz JF, Brown RJ, Oral EA. "Fat Shadows" From DXA for the Qualitative Assessment of Lipodystrophy: When a Picture Is Worth a Thousand Numbers. Diabetes Care. 2018 Oct;41(10):2255-2258. doi: 10.2337/dc18-0978.
Brown RJ, Meehan CA, Cochran E, Rother KI, Kleiner DE, Walter M, Gorden P. Effects of Metreleptin in Pediatric Patients With Lipodystrophy. J Clin Endocrinol Metab. 2017 May 1;102(5):1511-1519. doi: 10.1210/jc.2016-3628.

المصدر: An Open-label Extension of APG-20 Study to Evaluate the Long-term Safety and Efficacy of Daily Subcutaneous Metreleptin Treatment in Subjects With Partial Lipodystrophy

المصدر: A Pilot Study to Assess Feasibility of Adipose Tissue Triglyceride (TG) Labelling in Familial Partial Lipodystrophy (FPLD)

المؤلفون: Sorbonne University, University Hospital, Lille, University of Cambridge, Endocrinology Research Centre, Moscow, University of Leipzig, University of Amsterdam, Dokuz Eylul University, IRCCS Azienda Ospedaliero-Universitaria di Bologna, University of Rome Tor Vergata, University of Pisa, University of Santiago de Compostela, Università degli Studi del Piemonte Orientale Amedeo Avogadro, University Hospital Muenster, Szeged University, Centro Hospitalar do Porto, University Medical Centre Ljubljana, Medical University of Vienna, Ostschweizer Kinderspital

المساهمون: Julia von Schnurbein, Prinicipal Investigator

المصدر: Osse Registry for Patients With Lipodystrophy Run by the European Consortium of Lipodystrophies (ECLip)
von Schnurbein J, Adams C, Akinci B, Ceccarini G, D'Apice MR, Gambineri A, Hennekam RCM, Jeru I, Lattanzi G, Miehle K, Nagel G, Novelli G, Santini F, Santos Silva E, Savage DB, Sbraccia P, Schaaf J, Sorkina E, Tanteles G, Vantyghem MC, Vatier C, Vigouroux C, Vorona E, Araujo-Vilar D, Wabitsch M. European lipodystrophy registry: background and structure. Orphanet J Rare Dis. 2020 Jan 15;15(1):17. doi: 10.1186/s13023-020-1295-y.

Other URLs: https://www.eclip-web.org/information/registry/

المساهمون: Mandour Omer Mandour, Wellcome Trust Senior Clinical Fellow

المصدر: Evaluating the Therapeutic Efficacy and Metabolic Impact of a Low Energy Diet (LED) in People With Familial Partial Lipodystrophy and Diabetes
Lim EL, Hollingsworth KG, Aribisala BS, Chen MJ, Mathers JC, Taylor R. Reversal of type 2 diabetes: normalisation of beta cell function in association with decreased pancreas and liver triacylglycerol. Diabetologia. 2011 Oct;54(10):2506-14. doi: 10.1007/s00125-011-2204-7. Epub 2011 Jun 9.
Lean ME, Leslie WS, Barnes AC, Brosnahan N, Thom G, McCombie L, Peters C, Zhyzhneuskaya S, Al-Mrabeh A, Hollingsworth KG, Rodrigues AM, Rehackova L, Adamson AJ, Sniehotta FF, Mathers JC, Ross HM, McIlvenna Y, Stefanetti R, Trenell M, Welsh P, Kean S, Ford I, McConnachie A, Sattar N, Taylor R. Primary care-led weight management for remission of type 2 diabetes (DiRECT): an open-label, cluster-randomised trial. Lancet. 2018 Feb 10;391(10120):541-551. doi: 10.1016/S0140-6736(17)33102-1. Epub 2017 Dec 5.

المؤلفون: Rajan, Remya¹ (AUTHOR), Chapla, Aaron¹ (AUTHOR), Johnson, Jabasteen¹ (AUTHOR), Varghese, Deny¹ (AUTHOR), Asha, H. S.¹ (AUTHOR), Jebasingh, Felix¹ (AUTHOR), Kapoor, Nitin¹ (AUTHOR), Paul, Thomas V.¹ (AUTHOR), Thomas, Nihal¹ (AUTHOR) nihal_thomas@yahoo.com

المصدر: Scientific Reports. 11/16/2024, Vol. 14 Issue 1, p1-9. 9p.

مصطلحات موضوعية: *RESOURCE-limited settings, *LIPODYSTROPHY, *GENETIC testing, *GENETIC variation, *GLYCEMIC control

المؤلفون: Chng, Wei Qiang¹ (AUTHOR), Chen, Li Houng¹ (AUTHOR), Tang, Po Yin² (AUTHOR), Oh, Choon Chiat¹ (AUTHOR) oh.choon.chiat@singhealth.com.sg

المصدر: American Journal of Medicine. Dec2024, Vol. 137 Issue 12, pe235-e237. 3p.

مصطلحات موضوعية: *LYMPHOPROLIFERATIVE disorders, *LIPODYSTROPHY

المصدر: Natural History of Pregnancy and Pregnancy Outcomes in Metreleptin-Treated vs Untreated Subjects With Lipodystrophy

المصدر: A 12-Month Randomized, Multicenter, Double-Blind, Placebo-Controlled Phase 3 Study to Evaluate the Safety and Efficacy of Daily Subcutaneous Metreleptin Treatment in Subjects with Partial Lipodystrophy

المصدر: Compassionate Use Treatment Protocol I4V-MC-JAGA: Treatment of Conditions Expected to Benefit From JAK 1/2 Inhibition: CANDLE, CANDLE-Related Conditions, SAVI and Severe Juvenile Dermatomyositis
Adang LA, Gavazzi F, D'Aiello R, Isaacs D, Bronner N, Arici ZS, Flores Z, Jan A, Scher C, Sherbini O, Behrens EM, Goldbach-Mansky R, Olson TS, Lambert MP, Sullivan KE, Teachey DT, Witmer C, Vanderver A, Shults J. Hematologic abnormalities in Aicardi Goutieres Syndrome. Mol Genet Metab. 2022 Aug;136(4):324-329. doi: 10.1016/j.ymgme.2022.06.003. Epub 2022 Jun 16.
Kim H, Dill S, O'Brien M, Vian L, Li X, Manukyan M, Jain M, Adeojo LW, George J, Perez M, Grom AA, Sutter M, Feldman BM, Yao L, Millwood M, Brundidge A, Pichard DC, Cowen EW, Shi Y, Lu S, Tsai WL, Gadina M, Rider LG, Colbert RA. Janus kinase (JAK) inhibition with baricitinib in refractory juvenile dermatomyositis. Ann Rheum Dis. 2021 Mar;80(3):406-408. doi: 10.1136/annrheumdis-2020-218690. Epub 2020 Aug 25. No abstract available.
Sanchez GAM, Reinhardt A, Ramsey S, Wittkowski H, Hashkes PJ, Berkun Y, Schalm S, Murias S, Dare JA, Brown D, Stone DL, Gao L, Klausmeier T, Foell D, de Jesus AA, Chapelle DC, Kim H, Dill S, Colbert RA, Failla L, Kost B, O'Brien M, Reynolds JC, Folio LR, Calvo KR, Paul SM, Weir N, Brofferio A, Soldatos A, Biancotto A, Cowen EW, Digiovanna JJ, Gadina M, Lipton AJ, Hadigan C, Holland SM, Fontana J, Alawad AS, Brown RJ, Rother KI, Heller T, Brooks KM, Kumar P, Brooks SR, Waldman M, Singh HK, Nickeleit V, Silk M, Prakash A, Janes JM, Ozen S, Wakim PG, Brogan PA, Macias WL, Goldbach-Mansky R. JAK1/2 inhibition with baricitinib in the treatment of autoinflammatory interferonopathies. J Clin Invest. 2018 Jul 2;128(7):3041-3052. doi: 10.1172/JCI98814. Epub 2018 Jun 11.
Kim H, Brooks KM, Tang CC, Wakim P, Blake M, Brooks SR, Montealegre Sanchez GA, de Jesus AA, Huang Y, Tsai WL, Gadina M, Prakash A, Janes JM, Zhang X, Macias WL, Kumar P, Goldbach-Mansky R. Pharmacokinetics, Pharmacodynamics, and Proposed Dosing of the Oral JAK1 and JAK2 Inhibitor Baricitinib in Pediatric and Young Adult CANDLE and SAVI Patients. Clin Pharmacol Ther. 2018 Aug;104(2):364-373. doi: 10.1002/cpt.936. Epub 2017 Dec 8.

المصدر: Phase II Study of Growth Hormone Inhibition Using Pegvisomant In Severe Insulin Resistance

المصدر: A 24-Month, Multi-Centre, Open Label Phase IV Post Authorisation Efficacy Study to Evaluate the Efficacy, Safety and Immunogenicity of Daily Subcutaneous Metreleptin Treatment in Patients With Partial Lipodystrophy

المصدر: A Prospective, Open-label, Non-comparative, Multicentre, Post-market Clinical Follow-up Study of the Princess® VOLUME Performance and Safety for Correction of Facial Lipoatrophy, Morphological Asymmetry of the Face, or Debilitating Scars

المصدر: A Prospective, Open-label, Non-comparative, Multicentre, Post-market Clinical Follow-up Study of the Princess® FILLER Performance and Safety for Correction of Facial Lipoatrophy, Morphological Asymmetry of the Face, or Debilitating Scars

المصدر: A Randomized Double-Blind Placebo-Controlled Study of the LEPR Agonist Antibody REGN4461 for the Treatment of Metabolic Abnormalities in Patients With Familial Partial Lipodystrophy

Other URLs: https://vivli.org/

المصدر: A Randomized, Double-Blind, Placebo-Controlled Study of REGN4461, a Leptin Receptor Agonist Antibody, in Patients With Generalized Lipodystrophy

Other URLs: https://vivli.org/

المؤلفون: Patni, Nivedita¹ (AUTHOR), Chard, Craig² (AUTHOR), Araújo-Vilar, David³ (AUTHOR), Phillips, Helen⁴ (AUTHOR), Magee, David A.⁴ (AUTHOR) david.magee@chiesi.com, Akinci, Baris⁵ (AUTHOR)

المصدر: Orphanet Journal of Rare Diseases. 7/11/2024, Vol. 19 Issue 1, p1-11. 11p.

مصطلحات موضوعية: *PHYSICIANS' attitudes, *LIPODYSTROPHY, *PATIENTS' attitudes, *DELAYED diagnosis, *MEDICAL specialties & specialists

المؤلفون: da Silva, Monique Alvares¹ (NURSE), Soares, Reivla Marques Vasconcelos² (NURSE), de Oliveira Filho, Antônio Fernandes³ (AUTHOR), Campos, Leonardo René Santos⁴ (NURSE), de Lima, Josivan Gomes² (NURSE), de Melo Campos, Julliane Tamara Araújo¹ (NURSE) julliane.campos@ufrn.br

المصدر: Diabetology & Metabolic Syndrome. 7/1/2024, Vol. 16 Issue 1, p1-13. 13p.

مصطلحات موضوعية: *LIPODYSTROPHY, *TYPE 2 diabetes, *GENETIC variation, *ADIPOSE tissues, *INSULIN resistance, *PANCREATIC beta cells

مصطلحات جغرافية: BRAZIL