Publications and Presentations
Publications
Ervin, C., et al. Experiences of Hunger and Improved Quality of Life with Setmelanotide Treatment in Bardet-Biedl Syndrome. Advances in Therapy (2023) https://link.springer.com/article/10.1007/s12325-023-02443-y
Haqq, A., et al. Efficacy and safety of setmelanotide, a melanocortin-4 receptor agonist, in patients with Bardet-Biedl syndrome and Alström syndrome: a multicentre, randomised, double-blind, placebo-controlled, phase 3 trial with an open-label period. The Lancet Diabetes & Endocrinology. Online first (2022). https://doi.org/10.1016/S2213-8587(22)00277-7
Clément, K., et al. Efficacy and safety of setmelanotide, an MC4R agonist, in individuals with severe obesity due to LEPR or POMC deficiency: single-arm, open-label, multicentre, phase 3 trials. The Lancet Diabetes & Endocrinology. Online first (2020). https://doi.org/10.1016/S2213-8587(20)30364-8
Clément, K., et al. MC4R agonism promotes durable weight loss in patients with leptin receptor deficiency. Nat. Med. 24, 551–555 (2018). https://doi.org/10.1038/s41591-018-0015-9
Kühnen, P., et al. Proopiomelanocortin Deficiency Treated with a Melanocortin-4 Receptor Agonist. New England Journal of Medicine; 375:240-6. DOI: 10.1056/NEJMoa1512693(2016). https://www.nejm.org/doi/10.1056/NEJMoa1512693
Garfield, AS. et al. Role of central melanocortin pathways in energy homeostasis. Trends in Endocrinology and Metabolism: TEM, 203-215. DOI: 10.1016/j.tem.2009.02.002 (2009).https://doi.org/10.1016/j.tem.2009.02.002
To request a copy of an article, email [email protected].
Presentations
ESPE 2023 – European Society for Paediatric Endocrinology Annual Meeting
Frequency of Obesity-Related Gene Variants in a European Population With Early-Onset, Severe Obesity
Jesús Argente, MD, PhD, Professor in the Department of Pediatrics and Pediatric Endocrinology, Universidad Autónoma de Madrid
Slides
Impact of Setmelanotide on Metabolic Syndrome Risk in Pediatric Patients With POMC and LEPR Deficiency
Martin Wabitsch, M.D., Ph.D., Department of Pediatrics and Adolescent Medicine, University of Ulm in Germany
Slides
Impact of Setmelanotide on Future Metabolic Syndrome Risk in Pediatric Patients With Bardet-Biedl Syndrome
Jesús Argente, MD, PhD, Professor in the Department of Pediatrics and Pediatric Endocrinology, Universidad Autónoma de Madrid
Slides
Frequency of MC4R Pathway Variants in a European Cohort of Individuals With Early-Onset Severe Obesity
Anthony Goldstone, M.D., Ph.D., Department of Brain Sciences, Imperial College London
Slides
ENDO 2023 – Annual Conference
Weight Reduction After 6 Months Of Setmelanotide Treatment in Patients With Hypothalamic Obesity
Christian L. Roth, M.D., Seattle Children’s Research Institute, Seattle, WA
Poster
Treatment History and Comorbidities Reported by Patients With Hypothalamic Obesity Treated With Setmelanotide in a Phase 2 Trial
Christian L. Roth, M.D., Seattle Children’s Research Institute, Seattle, WA
Poster
Effect of Setmelanotide on Metabolic Parameters and Vital Signs in a Phase 2 Trial of Patients With Hypothalamic Obesity
Michael Gottschalk, M.D., Ph.D., Rady Children’s Hospital-San Diego, Professor, UC San Diego School of Medicine
Poster
Trial Design of a Double-blind, Randomized, Placebo-Controlled, Phase 3 Study of Setmelanotide in Patients With Hypothalamic Obesity
Ashley Shoemaker, M.D., Pediatric Endocrinology, Vanderbilt University Medical Center
Poster
The Obesity Society’s ObesityWeek® 2022
Efficacy and Safety Analysis of Setmelanotide As a Novel Treatment for Hypothalamic Obesity
Christian L. Roth, M.D., Seattle Children’s Research Institute, Seattle, WA
Poster
Substantial Burden Associated With Hyperphagia and Obesity in Children With Bardet-Biedl Syndrome
Jeremy Pomeroy, Ph.D., M.S., Marshfield Clinic Research Institute
Poster
Exploration of Clinical Improvements Following Setmelanotide Treatment in Patients With Bardet-Biedl Syndrome
Andrea Haqq, M.D., M.H.S., Division of Pediatric Endocrinology at the University of Alberta
Poster
Natural History of Weight Gain in Children With Bardet-Biedl Syndrome: Results From the Clinical Registry Investigating Bardet-Biedl Syndrome
Jeremy Pomeroy, Ph.D., M.S., Marshfield Clinic Research Institute
Poster
Weight Outcomes With Setmelanotide Over 3 Years in Patients With POMC or LEPR Deficiency Obesity
Karine Clément, M.D., Ph.D., Professor of Nutrition at Pitié-Salpêtrière Hospital, Sorbonne Université and Head of the INSERM Nutriomics Laboratory in Paris
Slides
Variants in Obesity-Related Genes in a Population With Early-Onset Obesity
Patrick Kleyn, Ph.D., SVP, Head of Translational Research and Development at Rhythm Pharmaceuticals
Poster
Frequency of MC4R Pathway Variants in a Large US Cohort of Patients With Severe Obesity
Elliot Bromberg, Beghou Consulting, Boston, MA
Poster
Clinical Benefit of Setmelanotide in Patients With Alström Syndrome
Andrea Haqq, M.D., M.H.S., Division of Pediatric Endocrinology at the University of Alberta
Poster
Effect of Long-term Setmelanotide Use on Body Composition in Patients With POMC or LEPR Deficiency Obesity
Karine Clément, M.D., Ph.D., Professor of Nutrition at Pitié-Salpêtrière Hospital, Sorbonne Université and Head of the Inserm Nutriomics Laboratory in Paris
Poster
Uncovering Rare Obesity Genetic Testing Program: Utility of Genetic Testing in Adults With Obesity
Patrick Kleyn, Ph.D., SVP, Head of Translational Research and Development at Rhythm Pharmaceuticals
Poster
Frequency of BBS and ALMS1 Variants in a Cohort With Early-Onset Obesity
Patrick Kleyn, Ph.D., SVP, Head of Translational Research and Development at Rhythm Pharmaceuticals
Poster
International Meeting on Pathway-Related Obesity: Vision of Excellence (IMPROVE) 2022
Setmelanotide in Patients with Obesity due to Certain MC4R Variants Stratified as Rescuable or Nonrescuable Based on an In Vitro Functional Assay
Poster
ESPE 2022 – European Society for Paediatric Endocrinology Annual Meeting
The Multifaceted Burden Experienced by Caregivers of Individuals with Bardet-Biedl Syndrome: Findings from the CARE-BBS Study
Elizabeth Forsythe, M.D., Ph.D., UCL Great Ormond Street Institute of Child Health, London.
Slides
Caregiver Burden in Bardet-Biedl Syndrome: a Survey of Obesity and Hyperphagia Impacts
Dr. Forsyth
Poster
Effects of Setmelanotide Treatment in Children and Adolescents with Proopiomelanocortin (POMC) Deficiency, Leptin Receptor (LEPR) Deficiency, and Bardet-Biedl Syndrome (BBS)
Jesús Argente, MD, Professor in the Department of Pediatrics and Pediatric Endocrinology, Universidad Autónoma de Madrid
Slides
VENTURE: Design of a Phase 3 Multicenter, 1-Year, Open-label Trial of Setmelanotide in Pediatric Patients Aged 2 to <6 Years with Rare Genetic Diseases of Obesity
Dr. Argente
Poster
ENDO 2022 – Annual Conference
Long-term Efficacy of Setmelanotide in Patients with Bardet-Biedl Syndrome
Jesús Argente, M.D., Ph.D., Universidad Autónoma de Madrid in Spain
Poster
Long-term Efficacy of Setmelanotide in Patients With POMC or LEPR Deficiency Obesity
Karine Clément, M.D., Ph.D., Professor of Nutrition at Pitié-Salpêtrière Hospital, Sorbonne Université and Head of the INSERM Nutriomics Laboratory in Paris
Poster
Body Mass Index and Weight Reduction in Patients with SH2B1 Deficiency Obesity After 1 Year of Setmelanotide
Sadaf Farooqi, M.D., Ph.D, the Wellcome-MRC Institute of Metabolic Science at the University of Cambridge
Poster
Body Mass Index and Weight Reductions in Patients with SRC1 Deficiency Obesity After 1 Year of Setmelanotide
Jesús Argente, M.D., Ph.D., Universidad Autónoma de Madrid in Spain.
Poster
Body Mass Index and Weight Reductions in Patients with Obesity Due to Heterozygous Variants in POMC, PCSK1, or LEPR After 1 Year of Setmelanotide
Brieana Buckley, PharmD, M.S., Vice President of Medical Affairs at Rhythm
Slides
Setmelanotide in Patients With Obesity Due to Heterozygous Variants in POMC, LEPR, NCOA1, or SH2B1 Genes: Design of EMANATE—a Placebo-Controlled Phase 3 Trial
Sadaf Farooqi, M.D., Ph.D, the Wellcome-MRC Institute of Metabolic Science at the University of Cambridge
Poster
