Setmelanotide in patients aged 2–5 years with rare MC4R pathway-associated obesity (VENTURE): a 1 year, open-label, multicenter, phase 3 trial
Abstract
Background Setmelanotide, a melanocortin-4 receptor (MC4R) agonist, has been shown to reduce hunger and weight in patients aged 6 years and older with proopiomelanocortin (POMC) deficiency (including biallelic variants in proprotein convertase subtilisin/kexin type 1 [PCSK1]), leptin receptor (LEPR) deficiency, or Bardet-Biedl syndrome (BBS). No approved therapies for patients younger than 6 years old currently exist. The phase 3, open-label VENTURE trial aimed to evaluate the efficacy and safety of setmelanotide in patients aged 2-5 years with POMC or LEPR deficiency or BBS. Methods This phase 3, open-label, multicentre trial, conducted across six sites in the USA, the UK, Spain, and Australia, enrolled eligible patients aged 2-5 years who had hyperphagia and obesity due to biallelic POMC (including PCSK1) or LEPR variants or genetically confirmed BBS. Open-label subcutaneous setmelanotide was administered once daily for 52 weeks, starting at 0·5 mg with doses increasing every 2 weeks in 0·5 mg increments until reaching the maximum dose based on weight. The co-primary endpoints at week 52 were the percentage of patients reaching a 0·2-point decrease or greater in BMI Z score (a statistical measure used to assess BMI in paediatric patients considering a patient's BMI and comparing it to reference values for the same age and sex) and mean percent change in BMI. Additional endpoints measured safety, hunger, weight-related outcomes, and caregiver burden. The study is registered at ClinicalTrials.gov (NCT04966741) and is complete. Findings Between March 8, 2022, and Sept 18, 2023, 13 patients were screened at the six sites, and 12 patients were enrolled in the study (seven with POMC or LEPR and five with BBS); one patient with BBS was excluded as their BMI was not at the 97th percentile or above. Of the 12 patients enrolled, most were male (seven [58%] vs five [42%] for female) and the mean age was 3·6 years (SD 0·9). 11 patients completed the trial. Ten (83%) of the 12 overall participants reached a 0·2-point reduction or more in BMI Z score per WHO methodology at week 52 (95% CI 58·7-99·8). The mean percent change in BMI from baseline at week 52 was -18% (SD 13) in the overall safety population. Mean percent change in BMI at week 52 was -26% (SD 11) in patients with POMC or LEPR deficiency and -10% (9) in patients with BBS. Mean reductions in secondary endpoints of BMI Z score (3·4 [2·5]) and percent of the BMI 95th percentile (32·5 [22·9]) were seen at Week 52. 91% of caregivers reported that patients were less hungry than at baseline. All adverse events were mild or moderate; skin hyperpigmentation, vomiting, nasopharyngitis, upper respiratory tract infection, and injection site reactions were most common. No serious adverse events or adverse events leading to study discontinuation or death were reported. Interpretation To our knowledge this is the first trial of setmelanotide in patients younger than 6 years old. These results support the benefit of the drug as an early intervention to manage obesity in this population. Funding Rhythm Pharmaceuticals.
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In the VENTURE Phase III trial of children aged 2 to under 5 with POMC or LEPR defects or BBS treated for 1 year, about 83% achieved a ≥0.2 reduction in BMI z-score.
“The study found that about 83% achieved a ≥ 0.2 reduction in BMI z‑score and mean BMI reductions of the 26% for patients with POMC/LEPR deficiencies and of the 10% for BBS.”
From Precision obesity medicine: A phenotype-guided framework for pharmacologic therapy across the lifespan (2025) · cites this paper as [104]
In the VENTURE trial, most patients experienced skin hyperpigmentation, which was the main cause of treatment discontinuation.
“Most of the patients experience skin hyperpigmentation that is the main cause of treatment discontinuation.”
From Precision obesity medicine: A phenotype-guided framework for pharmacologic therapy across the lifespan (2025) · cites this paper as [104]
In the VENTURE trial, hunger score improved significantly in about 91% of treated patients.
“The hunger score ameliorated significantly in about ~ 91% of the treated patients.”
From Precision obesity medicine: A phenotype-guided framework for pharmacologic therapy across the lifespan (2025) · cites this paper as [104]
In the VENTURE trial, mean BMI reduction was 26% for patients with POMC/LEPR deficiencies and 10% for BBS.
“The study found that about 83% achieved a ≥ 0.2 reduction in BMI z‑score and mean BMI reductions of the 26% for patients with POMC/LEPR deficiencies and of the 10% for BBS.”
From Precision obesity medicine: A phenotype-guided framework for pharmacologic therapy across the lifespan (2025) · cites this paper as [104]
The VENTURE trial extended these findings to children aged 2-5 years, including those with Bardet-Biedl syndrome, confirming sustained benefits in early childhood.
“The VENTURE trial extended these findings to children aged 2–5 years, including those with Bardet–Biedl syndrome, confirming sustained benefits in early childhood [104].”
From Precision obesity medicine: A phenotype-guided framework for pharmacologic therapy across the lifespan (2025) · cites this paper as [104]
Cites
Richards, S., Aziz, N., Bale, S., Bick, D., Das, S., Gastier-Foster, J., Grody, W. W., Hegde, M., Lyon, E., Spector, E., et al. (2015) · Genetics in Medicine · doi:10.1038/gim.2015.30 · Cited by 32,594 · see citing works
Approximate is Better than “Exact” for Interval Estimation of Binomial Proportions
Agresti, A., & Coull, B. A. (1998) · The American Statistician · doi:10.1080/00031305.1998.10480550 · Cited by 3,223 · see citing works
Styne, D. M., Arslanian, S. A., Connor, E. L., Farooqi, I. S., Murad, M. H., Silverstein, J. H., & Yanovski, J. A. (2017) · The Journal of Clinical Endocrinology & Metabolism · doi:10.1210/jc.2016-2573 · Cited by 1,322 · see citing works
Self-citationNew criteria for improved diagnosis of Bardet-Biedl syndrome: results of a population survey
Beales, P. L., Elcioglu, N. H., Woolf, A. S., Parker, D., & Flinter, F. A. (1999) · Journal of Medical Genetics · doi:10.1136/jmg.36.6.437 · Cited by 968 · see citing works
Screening for Obesity in Children and Adolescents
US Preventive Services Task Force, Grossman, D. C., Bibbins-Domingo, K. B., Curry, S. J., Barry, M. J., Davidson, K. W., Doubeni, C. A., Epling, J. W., Kemper, A. R., Krist, A. H., et al. (2017) · JAMA · doi:10.1001/jama.2017.6803 · Cited by 822 · see citing works
Forsythe, E. L., & Beales, P. L. (2012) · European Journal of Human Genetics · doi:10.1038/ejhg.2012.115 · Cited by 746 · see citing works
Clément, K., van den Akker, E., Argente, J., Bahm, A., Chung, W. K., Connors, H., De Waele, K., Farooqi, I. S., Gonneau-Lejeune, J., Gordon, G., et al. (2020) · The Lancet Diabetes & Endocrinology · doi:10.1016/s2213-8587(20)30364-8 · Cited by 492 · see citing works
Self-citationThe Hunger Genes: Pathways to Obesity
van der Klaauw, A. A., & Farooqi, I. S. (2015) · Cell · doi:10.1016/j.cell.2015.03.008 · Cited by 393 · see citing works
Self-citationRequirement of Bardet-Biedl syndrome proteins for leptin receptor signaling
Seo, S., Guo, D., Bugge, K., Morgan, D. A., Rahmouni, K., & Sheffield, V. C. (2009) · Human Molecular Genetics · doi:10.1093/hmg/ddp031 · Cited by 315 · see citing works
Mutations in ligands and receptors of the leptin–melanocortin pathway that lead to obesity
Farooqi, I. S., & O’Rahilly, S. (2008) · Nature Clinical Practice Endocrinology & Metabolism · doi:10.1038/ncpendmet0966 · Cited by 295 · see citing works
Self-citationRare Genetic Forms of Obesity: Clinical Approach and Current Treatments in 2016
Huvenne, H., Dubern, B., Clément, K., & Poitou, C. (2016) · Obesity Facts · doi:10.1159/000445061 · Cited by 279 · see citing works
The melanocortin pathway and control of appetite-progress and therapeutic implications
Baldini, G., & Phelan, K. D. (2019) · Journal of Endocrinology · doi:10.1530/joe-18-0596 · Cited by 279 · see citing works
Pediatric obesity—Long‐term consequences and effect of weight loss
Marcus, C., Danielsson, P., & Hagman, E. (2022) · Journal of Internal Medicine · doi:10.1111/joim.13547 · Cited by 267 · see citing works
Schreiner, A. S., Morimoto, T., Arai, Y., & Zarit, S. (2006) · Aging & Mental Health · doi:10.1080/13607860500312142 · Cited by 254 · see citing works
Haqq, A. M., Chung, W. K., Dollfus, H., Haws, R. M., Martos-Moreno, G. Á., Poitou, C., Yanovski, J. A., Mittleman, R. S., Yuan, G., Forsythe, E., et al. (2022) · The Lancet Diabetes & Endocrinology · doi:10.1016/s2213-8587(22)00277-7 · Cited by 206 · see citing works
Self-citationComplexity and Stigma of Pediatric Obesity
Haqq, A. M., Kebbe, M., Tan, Q., Manco, M., & Salas, X. R. (2021) · Childhood Obesity · doi:10.1089/chi.2021.0003 · Cited by 187 · see citing works
New advances in the genetics of early onset obesity
Farooqi, I. S., & O’Rahilly, S. (2005) · International Journal of Obesity · doi:10.1038/sj.ijo.0803056 · Cited by 89 · see citing works
Self-citationda Fonseca, A. C. P., Mastronardi, C. A., Johar, A., Arcos‐Burgos, M., & Paz-Filho, G. (2017) · Journal of Diabetes and its Complications · doi:10.1016/j.jdiacomp.2017.04.026 · Cited by 82 · see citing works
Ayers, K., Glicksberg, B. S., Garfield, A., Longerich, S., White, J., Yang, P., Du, L., Chittenden, T., Gulcher, J., Roy, S., et al. (2018) · Journal of Clinical Endocrinology and Metabolism · doi:10.1210/jc.2018-00258 · Cited by 82 · see citing works
Roth, C. L., Scimia, C., Shoemaker, A. H., Gottschalk, M., Miller, J., Yuan, G., Malhotra, S., & Abuzzahab, M. J. (2024) · The Lancet Diabetes & Endocrinology · doi:10.1016/s2213-8587(24)00087-1 · Cited by 76 · see citing works
Self-citationBardet‐Biedl syndrome: Weight patterns and genetics in a rare obesity syndrome
Pomeroy, J. J., Krentz, A. D., Richardson, J. G., Berg, R. L., VanWormer, J. J., & Haws, R. M. (2020) · Pediatric Obesity · doi:10.1111/ijpo.12703 · Cited by 72 · see citing works
Kohlsdorf, K., Nunziata, A., Funcke, J., Brandt, S., von Schnurbein, J., Vollbach, H., Lennerz, B. S., Fritsch, M., Greber‐Platzer, S., Fröhlich-Reiterer, E. E., et al. (2018) · International Journal of Obesity · doi:10.1038/s41366-018-0049-6 · Cited by 66 · see citing works
Natural History of Obesity Due to POMC, PCSK1, and LEPR Deficiency and the Impact of Setmelanotide
Wabitsch, M., Farooqi, I. S., Flück, C. E., Bratina, N., Mallya, U. G., Stewart, M. W., Garrison, J., van den Akker, E. L. T., & Kühnen, P. (2022) · Journal of the Endocrine Society · doi:10.1210/jendso/bvac057 · Cited by 64 · see citing works
Self-citationRare genetic forms of obesity: From gene to therapy
Clément, K., Mosbah, H., & Poitou, C. (2020) · Physiology & Behavior · doi:10.1016/j.physbeh.2020.113134 · Cited by 53 · see citing works
Kayadjanian, N., Vrana-Diaz, C. J., Bohonowych, J. E. S., Strong, T. V., Morin, J., Potvin, D., & Schwartz, L. (2021) · PLoS ONE · doi:10.1371/journal.pone.0248739 · Cited by 46 · see citing works
Saeed, S., Khanam, R., Janjua, Q. M., Manzoor, J., Ning, L., Hanook, S., Canouil, M., Ali, M. A., Ayesha, H., Khan, W. I., et al. (2023) · Cell Reports Medicine · doi:10.1016/j.xcrm.2023.101187 · Cited by 36 · see citing works
Self-citationWabitsch, M., Fehnel, S. E., Mallya, U. G., Sluga-O'Callaghan, M., Richardson, D., Price, M. A., & Kühnen, P. (2022) · Advances in Therapy · doi:10.1007/s12325-022-02059-8 · Cited by 34 · see citing works
Burden of hyperphagia and obesity in Bardet–Biedl syndrome: a multicountry survey
Forsythe, E. L., Mallya, U. G., Yang, M., Huber, C., Cala, M. L., Greatsinger, A., Hagopian, E., Pomeroy, J. J., & Haqq, A. M. (2023) · Orphanet Journal of Rare Diseases · doi:10.1186/s13023-023-02723-4 · Cited by 28 · see citing works
Ervin, C. M., Norcross, L., Mallya, U. G., Fehnel, S. E., Mittleman, R. S., Webster, M., Haqq, A. M., & Haws, R. M. (2023) · Advances in Therapy · doi:10.1007/s12325-023-02443-y · Cited by 23 · see citing works
The melanocortin‐4 receptor pathway and the emergence of precision medicine in obesity management
Fansa, S., & Acosta, A. J. (2024) · Diabetes Obesity and Metabolism · doi:10.1111/dom.15555 · Cited by 17 · see citing works
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Sorted by citations
Tuccinardi, D., Masi, D., Watanabe, M., Zanghi Buffi, V., De Domenico, F., Berti, S., Cipriani, V., Manco, M., Manfrini, S., & Pagotto, U. (2025) · Journal of Endocrinological Investigation · doi:10.1007/s40618-025-02700-7 · Cited by 29 · see citing works
Finkelberg, I., Polichronidou, I. M., Hühne, T., Brensing, P., Karaterzi, S., Jaegers, J., Gäckler, A., Pape, L., & Cetiner, M. (2025) · Orphanet Journal of Rare Diseases · doi:10.1186/s13023-025-03835-9 · Cited by 11 · see citing works
Updates on Anti-Obesity Medications in Children and Adolescents
Salama, M., Hassan, D., & Kumar, S. (2025) · Children · doi:10.3390/children12101390 · Cited by 7 · see citing works
Monogenic obesity due to MC4R deficiency: lessons from a multigenerational case
Giannopoulou, E. Z., Zorn, S., Schirmer, M., Brandt-Heunemann, S., von Schnurbein, J., Nestoris, C., Moawia, A., Siebert, R., Denzer, C., & Wabitsch, M. (2026) · Molecular and Cellular Pediatrics · doi:10.1186/s40348-025-00214-z · Cited by 6 · see citing works
Wang, J., Qu, J., Ye, M., Feng, R., Hui, X., Yang, X., Jin, J., Tong, Q., Zhang, X., & Wang, Y. (2025) · Journal of Pharmaceutical Analysis · doi:10.1016/j.jpha.2025.101416 · Cited by 6 · see citing works
Obesity and emerging intervention strategies: Mechanistic insights and novel drug targets
Feng, X., Li, S., Li, J., & Su, Z. (2025) · Chinese Medical Journal · doi:10.1097/cm9.0000000000003919 · Cited by 6 · see citing works
The genetics of obesity: aetiology, prevention and therapy
Bonnefond, A., Bruner, W. S., Grant, S. F. A., Morandi, A., & Froguel, P. (2026) · Nature Metabolism · doi:10.1038/s42255-026-01497-w · Cited by 5 · see citing works
Pomeroy, J. J., Okorie, U. C., Richardson, J. G., Faucher, A., Ionescu‐Ittu, R., Vekeman, F., Huber, C., & Haws, R. M. (2025) · Pediatric Obesity · doi:10.1111/ijpo.70026 · Cited by 5 · see citing works
Self-citationFrom genes to care: the expanding therapeutic horizon in rare genetic obesities
Chalopin, S., Guillon, E., Faucher, P., Dubern, B., Clément, K., & Poitou, C. (2025) · Obesity and Endocrinology · doi:10.1093/obendo/wjaf007 · Cited by 4 · see citing works
Setmelanotide-mediated MC4R activation improves hypothalamic obesity via CaMKK2/AMPK pathways
Peng, J., Ou, Y., Zhou, M., Wang, X., Zhang, X., Long, H., Wu, G., Che, M., Li, K., Yang, L., et al. (2026) · Frontiers in Pharmacology · doi:10.3389/fphar.2025.1730786 · Cited by 4 · see citing works
Setmelanotide for the treatment of severe early-childhood genetic obesity
Roth, C. L. (2024) · The Lancet Diabetes & Endocrinology · doi:10.1016/s2213-8587(24)00312-7 · Cited by 4 · see citing works
Classification of childhood obesity using longitudinal clinical body mass index and its validation
Ebrahim, N., Khadegi, A., Deng, S., Qian, K., Yao, Z., Thaker, S. K., May, B. L., Patibandala, N., López‐Pintado, S., & Thaker, V. (2025) · International Journal of Obesity · doi:10.1038/s41366-025-01836-z · Cited by 4 · see citing works
Tackling ciliary specialization to understand phenotypic variability in human primary ciliopathies
Bachmann‐Gagescu, R., & Sayer, J. A. (2025) · Journal of Cell Science · doi:10.1242/jcs.264177 · Cited by 4 · see citing works
Precision Prevention, Diagnostics, and Treatment of Obesity: Pipedream or Reality?
Woolf, E. K., Diktas, H. E., Acosta, A. J., Apolzan, J. W., Ard, J. D., Boyle, K. E., Gilbert, J. A., Katzmarzyk, P. T., Laurienti, P. J., Lobstein, T., et al. (2025) · Obesity · doi:10.1002/oby.70015 · Cited by 5 · see citing works
Ashlaw, E. F., Elfers, C., Chichura, K. S., Miranda, I. C., McGivney, A., Chepurny, O. G., Holz, G. G., Mullins, G., den Hartigh, L. J., Liu, Y., et al. (2025) · Metabolism · doi:10.1016/j.metabol.2025.156414 · Cited by 3 · see citing works
Prindle, C. R., Bennett, T. L., Rajewski, B. H., Kelley, K. J., Impastato, A. C., Potterfield, R., Marks, D. L., & Delev, J. Y. (2026) · Frontiers in Endocrinology · doi:10.3389/fendo.2026.1797586 · Cited by 3 · see citing works
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Martos‐Moreno, G. Á., Guijo, B., Tena‐Sempere, M., Chowen, J. A., & Argente, J. (2026) · Trends in Endocrinology and Metabolism · doi:10.1016/j.tem.2026.03.006 · Cited by 5 · see citing works
Self-citationManagement of Childhood Obesity
Gad, H., Dauleh, H., Mohammed, I., Malik, R. A., & Hussain, K. (2026) · International Journal of Molecular Sciences · doi:10.3390/ijms27083528 · Cited by 4 · see citing works
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Tomlinson, J. (2025) · Journal of Clinical Investigation · doi:10.1172/jci191822 · Cited by 3 · see citing works
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Clément, K., van den Akker, E. L. T., Argente, J., Beales, P. L., Cetiner, M., Dubern, B., Farooqi, I. S., Gouronc, A., Kühnen, P., Beyec, J. L., et al. (2025) · Clinical Obesity · doi:10.1111/cob.70029 · Cited by 2 · see citing works
Self-citationWitek, K., Wydra, K., Suder, A., & Filip, M. (2025) · Pharmacological Reports · doi:10.1007/s43440-025-00785-8 · Cited by 2 · see citing works
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Manoria, P. C. (2025) · Cureus · doi:10.7759/cureus.96713 · Cited by 2 · see citing works
Argente, J., Haqq, A. M., Schorfheide, J. L., Touchot, N., Huber, C., Wiedemann, U., Pomeroy, J. J., & the Phase 3 BBS Trial Investigators (2026) · Obesity · doi:10.1002/oby.70125 · Cited by 2 · see citing works
Self-citationAhmad, Y., Wasan, R., Perchik, J. D., & Nanda, N. C. (2026) · Journal of Clinical Medicine · doi:10.3390/jcm15124430 · Cited by 2 · see citing works
Sun, Y., Abed, M., & Sohouli, M. H. (2025) · Diabetology & Metabolic Syndrome · doi:10.1186/s13098-025-02071-2 · Cited by 1 · see citing works
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Berman, C., Kohn, B., & Wilson, D. P. (2026) · Journal of clinical lipidology · doi:10.1016/j.jacl.2025.07.004 · Cited by 3 · see citing works
Xie, J., Yang, Y., Chen, W., (45927), L. W., Weng, S., Lyu, Q., & Cao, G. (2026) · Current Obesity Reports · doi:10.1007/s13679-026-00722-7 · Cited by 1 · see citing works
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Serbis, A., Kantza, E., Garoufou, M., Christou, M., Siomou, E. C., & Tigas, S. K. (2026) · Endocrine · doi:10.1007/s12020-026-04696-3 · Cited by 1 · see citing works
Kondou, A., Siolos, P., Sotiriou, G., Agakidis, C., Dotis, J., Christoforidis, A., & Printza, N. (2026) · International Journal of Molecular Sciences · doi:10.3390/ijms27177740 · Cited by 1 · see citing works
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Shriyan, R., & Nanda, P. M. (2026) · Journal of Pediatric Endocrinology and Diabetes · doi:10.25259/jped_170_2026 · Cited by 1 · see citing works
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Chen, Z., Zhou, Y., Li, G., MUTHANNA, A., d’Arqom, A., Tham, C. L., Ho, Y.-C., & Lee, M. T. (2026) · Journal of Diabetes & Metabolic Disorders · doi:10.1007/s40200-026-02110-3
Dauleh, H., & Hussain, K. (2026) · International Journal of Molecular Sciences · doi:10.3390/ijms27209269
Salama, M., & Kumar, S. (2026) · Current Atherosclerosis Reports · doi:10.1007/s11883-026-01478-0
- Authors
- Argente, J., Verge, C. F., Okorie, U., Fennoy, I., Kelsey, M. M., Cokkinias, C., Scimia, C., Lee, H.-M., & Farooqi, I. S.
- Affiliations
- Instituto de Salud Carlos III (ES)
- UNSW Sydney (AU)
- Marshfield Clinic Research Foundation (US)
- Columbia University Irving Medical Center (US)
- Children's Hospital Colorado (US)
- Rhythm (United states) (US)
- University of Cambridge (GB)
- Corresponding author
- Argente, Jesús
- Venue
- The Lancet Diabetes & Endocrinology
- Publisher
- Elsevier BV
- Published
- 2025-01
- Volume
- 13
- Issue
- 1
- Pages
- 29-37
- ISSN
- 2213-8587 (ISSN-L)
- OpenAlex
- W4404311903
- Cited by
- 55
- References
- 30
- Citations by year
- 2026: 34 · 2025: 20 · 2024: 1
- FWCI
- 11.84
- Type
- journal article
- Language
- en
- Open access
- closed
- Licence
- Locations
- The Lancet Diabetes & Endocrinology (publishedVersion)
- PubMed (publishedVersion)
- Topics
- Life Sciences › Neuroscience › Endocrine and Autonomic Systems › Regulation of Appetite and Obesity
- Health Sciences › Medicine › Pharmacology › Pharmacology and Obesity Treatment
- Health Sciences › Medicine › Surgery › Bariatric Surgery and Outcomes
- Keywords
- setmelanotide, melanocortin-4 receptor agonist, childhood obesity, PCSK1, leptin receptor deficiency, Bardet-Biedl syndrome, hyperphagia, BMI z-score, hunger, caregiver burden, drug safety, open-label extension, dose escalation
- MeSH terms
- alpha-MSH, Child, Preschool, Female, Humans, Hyperphagia, Male, Obesity, Pro-Opiomelanocortin, Treatment Outcome, Receptor, Melanocortin, Type 4, Pediatric Obesity
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