Roth C, et al. Setmelanotide for the treatment of acquired hypothalamic obesity: a phase 2, open-label, multicentre trial. The Lancet Diabetes & Endocrinology. 2024;12(6):380-389.
Forsythe E, et al. Quality of life improvements following one year of setmelanotide in children and adult patients with Bardet–Biedl syndrome: phase 3 trial results. Orphanet J Rare Dis. 2023;18(1):12.
Shah BP, et al. Functional characterization of all missense variants in LEPR, PCSK1, and POMC genes arising from single-nucleotide variants. Expert Rev Endocrinol Metab. 2023;18(2):209-219.
Ervin C, et al. Interview-Based Patient- and Caregiver-Reported Experiences of Hunger and Improved Quality of Life with Setmelanotide Treatment in Bardet-Biedl Syndrome. Adv Ther. 2023;40(5):2394-2411.
Haqq AM, 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. Lancet Diabetes Endocrinol. 2022;10(12):859-868.
Kühnen P, et al. Quality of life outcomes in two phase 3 trials of setmelanotide in patients with obesity due to LEPR or POMC deficiency. Orphanet J Rare Dis. 2022;17(1):38.
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Clément K, et al. MC4R agonism promotes durable weight loss in patients with leptin receptor deficiency. Nat Med. 2018;24(5):551-555.
Kühnen P, et al. Proopiomelanocortin deficiency treated with a melanocortin-4 receptor agonist. N Engl J Med. 2016;375(3):240-246.
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ESPE 2024
Evaluating Setmelanotide Treatment for 12 Months in Pediatric Age Groups With Rare Melanocortin-4 Receptor Pathway–Related Obesity: Efficacy in Weight Reduction and Safety Outcomes
Dr. Erica L. T. van den Akker
Poster
3-Month Real-World Setmelanotide Hunger and Weight Outcomes in Four French Paediatric Patients with Acquired or Congenital Hypothalamic Obesity
Dr. Ahlam Azar-Kolakez
Poster
Frequency of Bardet-Biedl Syndrome Variants in a Population with Early-Onset Obesity
Dr. Jesús Argente
Presentation
Frequency of Rare Syndromic Diseases in a Population with Early-Onset Obesity
Dr. Melania Manco
Presentation
Frequency of Rare Non-Syndromic Diseases in a Population with Early-Onset Obesity
Dr. Jesús Argente
Presentation
TOS 2024
Weight Loss at 18 Months of Setmelanotide in 2- to <6-Year-Old Patients With Rare MC4R Pathway Diseases
Dr. Jesús Argente
Poster
3-Month Real-World Setmelanotide Hunger and Weight Outcomes in Patients with Hypothalamic Obesity
Dr. Blandine Gatta-Cherifi
Poster
DAYBREAK Trial: Setmelanotide Versus Placebo in Patients With Melanocortin-4 Receptor Pathway Variants
Dr. Gloria Ortiz
Poster
Body Composition Improvements After 12 Months of Setmelanotide in Acquired Hypothalamic Obesity
Dr. Christian L. Roth
Poster
Risk Factors Correlating With MC4R Pathway Variants on Genetic Testing
Patrick Sleiman
Poster
ENDO 2024
Patient- and Caregiver-Reported Experiences of Hunger, Weight, and Energy in Acquired Hypothalamic Obesity Before and During Setmelanotide Treatment
Dr. Christian Roth
Poster
Clinical Characteristics of Patients With Obesity Secondary to Different MC4R Pathway Diseases and 1-Year Response to Setmelanotide
Dr. Christian Roth
Poster
Hyperphagia and Body Weight Gain Are Reduced by Once-Weekly Treatment With RM-718, a Selective Melanocortin-4 Receptor Agonist, in Zucker Obese Rats
Dr. Danica Grujic
Presentation
RESTORE: Real-world Study of Setmelanotide in Patients With MC4R Pathway Diseases
Caroline Huber
Poster
Design of a Phase 1 Trial of Once-Weekly RM-718, a Selective Melanocortin-4 Receptor Agonist, in Patients With General or Hypothalamic Obesity
Dr. Christian Roth
Poster
Stage 1 Results of the Phase 2 DAYBREAK Trial Assessing Setmelanotide in Patients With Early-Onset, Severe Obesity and Hyperphagia Who Carried a Confirmed Variant Related to the Melanocortin-4 Receptor Pathway
Dr. Elif Oral
Poster
PES 2024
Weight-Related Measures in Pediatric Patients With Hypothalamic Obesity Treated With Setmelanotide for 12 Months
Jennifer Abuzzahab, M.D., Pediatric Endocrinology, Children’s Minnesota
Poster
Efficacy in Weight Reduction and Safety in Pediatric Age Groups With Rare Melanocortin-4 Receptor Pathway–Related Obesity Treated With Setmelanotide for 12 Months
Ashley Shoemaker, M.D., Pediatric Endocrinology, Vanderbilt University Medical Center
Poster
Efficacy and Safety of Setmelanotide in Patients Aged 2 to <6 Years With Rare Melanocortin-4 Receptor Pathway Diseases of Obesity: Results From VENTURE, a Phase 3, Multicenter, 1-Year, Open-Label Trial
Megan Kelsey, M.D., Children’s Hospital Colorado
Presentation
The Obesity Society’s ObesityWeek® 2023
Weight Reduction in Patients With Hypothalamic Obesity Treated With Setmelanotide for 12 Months
Christian L. Roth, M.D., Seattle Children’s Research Institute, Seattle, WA
Poster
3-Year Setmelanotide Weight Outcomes in Patients With Bardet-Biedl Syndrome and Obesity
Jack A. Yanovski, M.D., Ph.D., Eunice Kennedy Shriver National Institute of Child Health and Human Development, National Institutes of Health, Bethesda, MD
Poster
Cardiac, Renal, and Endocrine/Diabetes Mellitus Outcomes in Children With Bardet-Biedl Syndrome
Jeremy Pomeroy, Ph.D., M.S., Marshfield Clinic Research Institute
Poster
Impact of Setmelanotide on Metabolic Syndrome Risk in Patients With Bardet-Biedl Syndrome
Andrea Haqq, M.D., M.H.S., Division of Pediatric Endocrinology at the University of Alberta
Presentation
4-Year Setmelanotide Weight Outcomes in Patients With POMC and LEPR Deficiency Obesity
Wendy K. Chung, M.D., Ph.D., Division of Molecular Genetics, Department of Pediatrics, Columbia University, New York, NY
Presentation
Impact of Setmelanotide on Metabolic Syndrome Risk in Patients With POMC and LEPR Deficiency
Martin Wabitsch, M.D., Ph.D., Department of Pediatrics and Adolescent Medicine, University of Ulm in Germany
Presentation
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, M.D., Ph.D., 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, M.D., Ph.D., 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
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, M.D., 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
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