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Aniridia-ptosis-intellectual disability-familial obesity syndrome

MedGen UID:
929405
Concept ID:
C4303736
Disease or Syndrome
Synonyms: aniridia - ptosis - intellectual disability - familial obesity; Aniridia, ptosis, intellectual disability, familial obesity syndrome; aniridia-ptosis-intellectual disability-familial obesity syndrome
SNOMED CT: Aniridia, ptosis, intellectual disability, familial obesity syndrome (720987001)
Modes of inheritance:
Autosomal dominant inheritance
MedGen UID:
141047
Concept ID:
C0443147
Intellectual Product
Source: Orphanet
A mode of inheritance that is observed for traits related to a gene encoded on one of the autosomes (i.e., the human chromosomes 1-22) in which a trait manifests in heterozygotes. In the context of medical genetics, an autosomal dominant disorder is caused when a single copy of the mutant allele is present. Males and females are affected equally, and can both transmit the disorder with a risk of 50% for each child of inheriting the mutant allele.
 
Monarch Initiative: MONDO:0015198
Orphanet: ORPHA1067

Definition

An extremely rare syndrome described in three members of a family (a mother and her two children) with the association of various ocular abnormalities (partial or complete aniridia, ptosis, pendular nystagmus, corneal pannus, persistent pupillary membrane, lenticular opacities, foveal hypoplasia and low visual acuity) with various systemic anomalies including intellectual disability and obesity in the two children and alopecia, cardiac abnormalities and frequent spontaneous abortion in the mother. There have been no further descriptions in the literature since 1986. [from SNOMEDCT_US]

Term Hierarchy

CClinical test,  RResearch test,  OOMIM,  GGeneReviews,  VClinVar  
  • CROGVAniridia-ptosis-intellectual disability-familial obesity syndrome

Professional guidelines

PubMed

Joglar JA, Chung MK, Armbruster AL, Benjamin EJ, Chyou JY, Cronin EM, Deswal A, Eckhardt LL, Goldberger ZD, Gopinathannair R, Gorenek B, Hess PL, Hlatky M, Hogan G, Ibeh C, Indik JH, Kido K, Kusumoto F, Link MS, Linta KT, Marcus GM, McCarthy PM, Patel N, Patton KK, Perez MV, Piccini JP, Russo AM, Sanders P, Streur MM, Thomas KL, Times S, Tisdale JE, Valente AM, Van Wagoner DR; Peer Review Committee Members
Circulation 2024 Jan 2;149(1):e1-e156. Epub 2023 Nov 30 doi: 10.1161/CIR.0000000000001193. PMID: 38033089Free PMC Article
Nierenberg AA, Agustini B, Köhler-Forsberg O, Cusin C, Katz D, Sylvia LG, Peters A, Berk M
JAMA 2023 Oct 10;330(14):1370-1380. doi: 10.1001/jama.2023.18588. PMID: 37815563
Teede HJ, Tay CT, Laven JJE, Dokras A, Moran LJ, Piltonen TT, Costello MF, Boivin J, Redman LM, Boyle JA, Norman RJ, Mousa A, Joham AE
J Clin Endocrinol Metab 2023 Sep 18;108(10):2447-2469. doi: 10.1210/clinem/dgad463. PMID: 37580314Free PMC Article

Recent clinical studies

Therapy

Gu Y, Zhou G, Zhou F, Wu Q, Ma C, Zhang Y, Ding J, Hua K
Front Endocrinol (Lausanne) 2022;13:808898. Epub 2022 Apr 13 doi: 10.3389/fendo.2022.808898. PMID: 35498415Free PMC Article
Ryan DH
Handb Exp Pharmacol 2022;274:387-414. doi: 10.1007/164_2021_560. PMID: 34783910
Green M, Arora K, Prakash S
Int J Mol Sci 2020 Apr 21;21(8) doi: 10.3390/ijms21082890. PMID: 32326175Free PMC Article
Broughton DE, Moley KH
Fertil Steril 2017 Apr;107(4):840-847. Epub 2017 Mar 11 doi: 10.1016/j.fertnstert.2017.01.017. PMID: 28292619
Garvey WT, Mechanick JI, Brett EM, Garber AJ, Hurley DL, Jastreboff AM, Nadolsky K, Pessah-Pollack R, Plodkowski R; Reviewers of the AACE/ACE Obesity Clinical Practice Guidelines
Endocr Pract 2016 Jul;22 Suppl 3:1-203. Epub 2016 May 24 doi: 10.4158/EP161365.GL. PMID: 27219496

Prognosis

Mambrini SP, Menichetti F, Ravella S, Pellizzari M, De Amicis R, Foppiani A, Battezzati A, Bertoli S, Leone A
Nutrients 2023 May 31;15(11) doi: 10.3390/nu15112583. PMID: 37299546Free PMC Article
Spaander MCW, Zauber AG, Syngal S, Blaser MJ, Sung JJ, You YN, Kuipers EJ
Nat Rev Dis Primers 2023 Apr 27;9(1):21. doi: 10.1038/s41572-023-00432-7. PMID: 37105987Free PMC Article
Fan JG, Kim SU, Wong VW
J Hepatol 2017 Oct;67(4):862-873. Epub 2017 Jun 19 doi: 10.1016/j.jhep.2017.06.003. PMID: 28642059
Younossi ZM, Koenig AB, Abdelatif D, Fazel Y, Henry L, Wymer M
Hepatology 2016 Jul;64(1):73-84. Epub 2016 Feb 22 doi: 10.1002/hep.28431. PMID: 26707365
Bardou M, Barkun AN, Martel M
Gut 2013 Jun;62(6):933-47. Epub 2013 Mar 12 doi: 10.1136/gutjnl-2013-304701. PMID: 23481261

Clinical prediction guides

Marinelli S, Napoletano G, Straccamore M, Basile G
Acta Biomed 2022 Aug 31;93(4):e2022278. doi: 10.23750/abm.v93i4.13466. PMID: 36043953Free PMC Article
Litwin M, Kułaga Z
Pediatr Nephrol 2021 Apr;36(4):825-837. Epub 2020 May 9 doi: 10.1007/s00467-020-04579-3. PMID: 32388582Free PMC Article
Aron-Wisnewsky J, Clément K, Nieuwdorp M
Curr Diab Rep 2019 Jun 27;19(8):51. doi: 10.1007/s11892-019-1180-z. PMID: 31250122
Donini LM, Savina C, Coletti C, Paolini M, de Felice MR, del Balzo V, Cannella C, Scavone L
Ann Ig 2010 Nov-Dec;22(6):499-511. PMID: 21417168
Pan WH, Yeh WT
Asia Pac J Clin Nutr 2008;17(3):370-4. PMID: 18818155

Recent systematic reviews

Bukavina L, Bensalah K, Bray F, Carlo M, Challacombe B, Karam JA, Kassouf W, Mitchell T, Montironi R, O'Brien T, Panebianco V, Scelo G, Shuch B, van Poppel H, Blosser CD, Psutka SP
Eur Urol 2022 Nov;82(5):529-542. Epub 2022 Sep 10 doi: 10.1016/j.eururo.2022.08.019. PMID: 36100483
Gu Y, Zhou G, Zhou F, Wu Q, Ma C, Zhang Y, Ding J, Hua K
Front Endocrinol (Lausanne) 2022;13:808898. Epub 2022 Apr 13 doi: 10.3389/fendo.2022.808898. PMID: 35498415Free PMC Article
O'Sullivan DE, Sutherland RL, Town S, Chow K, Fan J, Forbes N, Heitman SJ, Hilsden RJ, Brenner DR
Clin Gastroenterol Hepatol 2022 Jun;20(6):1229-1240.e5. Epub 2021 Jan 29 doi: 10.1016/j.cgh.2021.01.037. PMID: 33524598
Safaei M, Sundararajan EA, Driss M, Boulila W, Shapi'i A
Comput Biol Med 2021 Sep;136:104754. Epub 2021 Aug 16 doi: 10.1016/j.compbiomed.2021.104754. PMID: 34426171
Carbone A, Al Salhi Y, Tasca A, Palleschi G, Fuschi A, De Nunzio C, Bozzini G, Mazzaferro S, Pastore AL
Minerva Urol Nefrol 2018 Aug;70(4):393-400. Epub 2018 May 31 doi: 10.23736/S0393-2249.18.03113-2. PMID: 29856171

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