U.S. flag

An official website of the United States government

Format

Send to:

Choose Destination

Rare disease with autism

MedGen UID:
1843233
Concept ID:
C5680471
Disease or Syndrome
Orphanet: ORPHA180772

Professional guidelines

PubMed

Specchio N, Nabbout R, Aronica E, Auvin S, Benvenuto A, de Palma L, Feucht M, Jansen F, Kotulska K, Sarnat H, Lagae L, Jozwiak S, Curatolo P
Eur J Paediatr Neurol 2023 Nov;47:25-34. Epub 2023 Aug 30 doi: 10.1016/j.ejpn.2023.08.005. PMID: 37669572
Duis J, Nespeca M, Summers J, Bird L, Bindels-de Heus KGCB, Valstar MJ, de Wit MY, Navis C, Ten Hooven-Radstaake M, van Iperen-Kolk BM, Ernst S, Dendrinos M, Katz T, Diaz-Medina G, Katyayan A, Nangia S, Thibert R, Glaze D, Keary C, Pelc K, Simon N, Sadhwani A, Heussler H, Wheeler A, Woeber C, DeRamus M, Thomas A, Kertcher E, DeValk L, Kalemeris K, Arps K, Baym C, Harris N, Gorham JP, Bohnsack BL, Chambers RC, Harris S, Chambers HG, Okoniewski K, Jalazo ER, Berent A, Bacino CA, Williams C, Anderson A
Mol Genet Genomic Med 2022 Mar;10(3):e1843. Epub 2022 Feb 11 doi: 10.1002/mgg3.1843. PMID: 35150089Free PMC Article
Zollino M, Zweier C, Van Balkom ID, Sweetser DA, Alaimo J, Bijlsma EK, Cody J, Elsea SH, Giurgea I, Macchiaiolo M, Smigiel R, Thibert RL, Benoist I, Clayton-Smith J, De Winter CF, Deckers S, Gandhi A, Huisman S, Kempink D, Kruisinga F, Lamacchia V, Marangi G, Menke L, Mulder P, Nordgren A, Renieri A, Routledge S, Saunders CJ, Stembalska A, Van Balkom H, Whalen S, Hennekam RC
Clin Genet 2019 Apr;95(4):462-478. Epub 2019 Feb 18 doi: 10.1111/cge.13506. PMID: 30677142

Recent clinical studies

Etiology

Fu JM, Satterstrom FK, Peng M, Brand H, Collins RL, Dong S, Wamsley B, Klei L, Wang L, Hao SP, Stevens CR, Cusick C, Babadi M, Banks E, Collins B, Dodge S, Gabriel SB, Gauthier L, Lee SK, Liang L, Ljungdahl A, Mahjani B, Sloofman L, Smirnov AN, Barbosa M, Betancur C, Brusco A, Chung BHY, Cook EH, Cuccaro ML, Domenici E, Ferrero GB, Gargus JJ, Herman GE, Hertz-Picciotto I, Maciel P, Manoach DS, Passos-Bueno MR, Persico AM, Renieri A, Sutcliffe JS, Tassone F, Trabetti E, Campos G, Cardaropoli S, Carli D, Chan MCY, Fallerini C, Giorgio E, Girardi AC, Hansen-Kiss E, Lee SL, Lintas C, Ludena Y, Nguyen R, Pavinato L, Pericak-Vance M, Pessah IN, Schmidt RJ, Smith M, Costa CIS, Trajkova S, Wang JYT, Yu MHC; Autism Sequencing Consortium (ASC); Broad Institute Center for Common Disease Genomics (Broad-CCDG); iPSYCH-BROAD Consortium, Cutler DJ, De Rubeis S, Buxbaum JD, Daly MJ, Devlin B, Roeder K, Sanders SJ, Talkowski ME
Nat Genet 2022 Sep;54(9):1320-1331. Epub 2022 Aug 18 doi: 10.1038/s41588-022-01104-0. PMID: 35982160Free PMC Article
Zhou X, Feliciano P, Shu C, Wang T, Astrovskaya I, Hall JB, Obiajulu JU, Wright JR, Murali SC, Xu SX, Brueggeman L, Thomas TR, Marchenko O, Fleisch C, Barns SD, Snyder LG, Han B, Chang TS, Turner TN, Harvey WT, Nishida A, O'Roak BJ, Geschwind DH; SPARK Consortium, Michaelson JJ, Volfovsky N, Eichler EE, Shen Y, Chung WK
Nat Genet 2022 Sep;54(9):1305-1319. Epub 2022 Aug 18 doi: 10.1038/s41588-022-01148-2. PMID: 35982159Free PMC Article
Thapar A, Rutter M
J Autism Dev Disord 2021 Dec;51(12):4321-4332. doi: 10.1007/s10803-020-04685-z. PMID: 32940822Free PMC Article
Satterstrom FK, Kosmicki JA, Wang J, Breen MS, De Rubeis S, An JY, Peng M, Collins R, Grove J, Klei L, Stevens C, Reichert J, Mulhern MS, Artomov M, Gerges S, Sheppard B, Xu X, Bhaduri A, Norman U, Brand H, Schwartz G, Nguyen R, Guerrero EE, Dias C; Autism Sequencing Consortium; iPSYCH-Broad Consortium, Betancur C, Cook EH, Gallagher L, Gill M, Sutcliffe JS, Thurm A, Zwick ME, Børglum AD, State MW, Cicek AE, Talkowski ME, Cutler DJ, Devlin B, Sanders SJ, Roeder K, Daly MJ, Buxbaum JD
Cell 2020 Feb 6;180(3):568-584.e23. Epub 2020 Jan 23 doi: 10.1016/j.cell.2019.12.036. PMID: 31981491Free PMC Article
Lai MC, Lombardo MV, Baron-Cohen S
Lancet 2014 Mar 8;383(9920):896-910. Epub 2013 Sep 26 doi: 10.1016/S0140-6736(13)61539-1. PMID: 24074734

Diagnosis

Boniel S, Szymańska K, Śmigiel R, Szczałuba K
Genes (Basel) 2021 Mar 25;12(4) doi: 10.3390/genes12040468. PMID: 33805950Free PMC Article
Thapar A, Rutter M
J Autism Dev Disord 2021 Dec;51(12):4321-4332. doi: 10.1007/s10803-020-04685-z. PMID: 32940822Free PMC Article
Scheffer IE, Nabbout R
Epilepsia 2019 Dec;60 Suppl 3:S17-S24. doi: 10.1111/epi.16386. PMID: 31904117
Marucci S, Ragione LD, De Iaco G, Mococci T, Vicini M, Guastamacchia E, Triggiani V
Endocr Metab Immune Disord Drug Targets 2018;18(4):316-324. doi: 10.2174/1871530318666180213111637. PMID: 29437020
Lai MC, Lombardo MV, Baron-Cohen S
Lancet 2014 Mar 8;383(9920):896-910. Epub 2013 Sep 26 doi: 10.1016/S0140-6736(13)61539-1. PMID: 24074734

Therapy

Specchio N, Nabbout R, Aronica E, Auvin S, Benvenuto A, de Palma L, Feucht M, Jansen F, Kotulska K, Sarnat H, Lagae L, Jozwiak S, Curatolo P
Eur J Paediatr Neurol 2023 Nov;47:25-34. Epub 2023 Aug 30 doi: 10.1016/j.ejpn.2023.08.005. PMID: 37669572
Neul JL, Percy AK, Benke TA, Berry-Kravis EM, Glaze DG, Marsh ED, Lin T, Stankovic S, Bishop KM, Youakim JM
Nat Med 2023 Jun;29(6):1468-1475. Epub 2023 Jun 8 doi: 10.1038/s41591-023-02398-1. PMID: 37291210Free PMC Article
Curatolo P, Specchio N, Aronica E
Lancet Neurol 2022 Sep;21(9):843-856. doi: 10.1016/S1474-4422(22)00213-7. PMID: 35963265
Johannesen KM, Liu Y, Koko M, Gjerulfsen CE, Sonnenberg L, Schubert J, Fenger CD, Eltokhi A, Rannap M, Koch NA, Lauxmann S, Krüger J, Kegele J, Canafoglia L, Franceschetti S, Mayer T, Rebstock J, Zacher P, Ruf S, Alber M, Sterbova K, Lassuthová P, Vlckova M, Lemke JR, Platzer K, Krey I, Heine C, Wieczorek D, Kroell-Seger J, Lund C, Klein KM, Au PYB, Rho JM, Ho AW, Masnada S, Veggiotti P, Giordano L, Accorsi P, Hoei-Hansen CE, Striano P, Zara F, Verhelst H, Verhoeven JS, Braakman HMH, van der Zwaag B, Harder AVE, Brilstra E, Pendziwiat M, Lebon S, Vaccarezza M, Le NM, Christensen J, Grønborg S, Scherer SW, Howe J, Fazeli W, Howell KB, Leventer R, Stutterd C, Walsh S, Gerard M, Gerard B, Matricardi S, Bonardi CM, Sartori S, Berger A, Hoffman-Zacharska D, Mastrangelo M, Darra F, Vøllo A, Motazacker MM, Lakeman P, Nizon M, Betzler C, Altuzarra C, Caume R, Roubertie A, Gélisse P, Marini C, Guerrini R, Bilan F, Tibussek D, Koch-Hogrebe M, Perry MS, Ichikawa S, Dadali E, Sharkov A, Mishina I, Abramov M, Kanivets I, Korostelev S, Kutsev S, Wain KE, Eisenhauer N, Wagner M, Savatt JM, Müller-Schlüter K, Bassan H, Borovikov A, Nassogne MC, Destrée A, Schoonjans AS, Meuwissen M, Buzatu M, Jansen A, Scalais E, Srivastava S, Tan WH, Olson HE, Loddenkemper T, Poduri A, Helbig KL, Helbig I, Fitzgerald MP, Goldberg EM, Roser T, Borggraefe I, Brünger T, May P, Lal D, Lederer D, Rubboli G, Heyne HO, Lesca G, Hedrich UBS, Benda J, Gardella E, Lerche H, Møller RS
Brain 2022 Sep 14;145(9):2991-3009. doi: 10.1093/brain/awab321. PMID: 34431999Free PMC Article
Taylor B, Miller E, Farrington CP, Petropoulos MC, Favot-Mayaud I, Li J, Waight PA
Lancet 1999 Jun 12;353(9169):2026-9. doi: 10.1016/s0140-6736(99)01239-8. PMID: 10376617

Prognosis

Tandon R, Nasrallah H, Akbarian S, Carpenter WT Jr, DeLisi LE, Gaebel W, Green MF, Gur RE, Heckers S, Kane JM, Malaspina D, Meyer-Lindenberg A, Murray R, Owen M, Smoller JW, Yassin W, Keshavan M
Schizophr Res 2024 Feb;264:1-28. Epub 2023 Dec 12 doi: 10.1016/j.schres.2023.11.015. PMID: 38086109
Jaganathan K, Kyriazopoulou Panagiotopoulou S, McRae JF, Darbandi SF, Knowles D, Li YI, Kosmicki JA, Arbelaez J, Cui W, Schwartz GB, Chow ED, Kanterakis E, Gao H, Kia A, Batzoglou S, Sanders SJ, Farh KK
Cell 2019 Jan 24;176(3):535-548.e24. Epub 2019 Jan 17 doi: 10.1016/j.cell.2018.12.015. PMID: 30661751
Marucci S, Ragione LD, De Iaco G, Mococci T, Vicini M, Guastamacchia E, Triggiani V
Endocr Metab Immune Disord Drug Targets 2018;18(4):316-324. doi: 10.2174/1871530318666180213111637. PMID: 29437020
Lai MC, Lombardo MV, Baron-Cohen S
Lancet 2014 Mar 8;383(9920):896-910. Epub 2013 Sep 26 doi: 10.1016/S0140-6736(13)61539-1. PMID: 24074734
Thapar A, Cooper M, Eyre O, Langley K
J Child Psychol Psychiatry 2013 Jan;54(1):3-16. Epub 2012 Sep 11 doi: 10.1111/j.1469-7610.2012.02611.x. PMID: 22963644Free PMC Article

Clinical prediction guides

Trost B, Thiruvahindrapuram B, Chan AJS, Engchuan W, Higginbotham EJ, Howe JL, Loureiro LO, Reuter MS, Roshandel D, Whitney J, Zarrei M, Bookman M, Somerville C, Shaath R, Abdi M, Aliyev E, Patel RV, Nalpathamkalam T, Pellecchia G, Hamdan O, Kaur G, Wang Z, MacDonald JR, Wei J, Sung WWL, Lamoureux S, Hoang N, Selvanayagam T, Deflaux N, Geng M, Ghaffari S, Bates J, Young EJ, Ding Q, Shum C, D'Abate L, Bradley CA, Rutherford A, Aguda V, Apresto B, Chen N, Desai S, Du X, Fong MLY, Pullenayegum S, Samler K, Wang T, Ho K, Paton T, Pereira SL, Herbrick JA, Wintle RF, Fuerth J, Noppornpitak J, Ward H, Magee P, Al Baz A, Kajendirarajah U, Kapadia S, Vlasblom J, Valluri M, Green J, Seifer V, Quirbach M, Rennie O, Kelley E, Masjedi N, Lord C, Szego MJ, Zawati MH, Lang M, Strug LJ, Marshall CR, Costain G, Calli K, Iaboni A, Yusuf A, Ambrozewicz P, Gallagher L, Amaral DG, Brian J, Elsabbagh M, Georgiades S, Messinger DS, Ozonoff S, Sebat J, Sjaarda C, Smith IM, Szatmari P, Zwaigenbaum L, Kushki A, Frazier TW, Vorstman JAS, Fakhro KA, Fernandez BA, Lewis MES, Weksberg R, Fiume M, Yuen RKC, Anagnostou E, Sondheimer N, Glazer D, Hartley DM, Scherer SW
Cell 2022 Nov 10;185(23):4409-4427.e18. doi: 10.1016/j.cell.2022.10.009. PMID: 36368308Free PMC Article
Fu JM, Satterstrom FK, Peng M, Brand H, Collins RL, Dong S, Wamsley B, Klei L, Wang L, Hao SP, Stevens CR, Cusick C, Babadi M, Banks E, Collins B, Dodge S, Gabriel SB, Gauthier L, Lee SK, Liang L, Ljungdahl A, Mahjani B, Sloofman L, Smirnov AN, Barbosa M, Betancur C, Brusco A, Chung BHY, Cook EH, Cuccaro ML, Domenici E, Ferrero GB, Gargus JJ, Herman GE, Hertz-Picciotto I, Maciel P, Manoach DS, Passos-Bueno MR, Persico AM, Renieri A, Sutcliffe JS, Tassone F, Trabetti E, Campos G, Cardaropoli S, Carli D, Chan MCY, Fallerini C, Giorgio E, Girardi AC, Hansen-Kiss E, Lee SL, Lintas C, Ludena Y, Nguyen R, Pavinato L, Pericak-Vance M, Pessah IN, Schmidt RJ, Smith M, Costa CIS, Trajkova S, Wang JYT, Yu MHC; Autism Sequencing Consortium (ASC); Broad Institute Center for Common Disease Genomics (Broad-CCDG); iPSYCH-BROAD Consortium, Cutler DJ, De Rubeis S, Buxbaum JD, Daly MJ, Devlin B, Roeder K, Sanders SJ, Talkowski ME
Nat Genet 2022 Sep;54(9):1320-1331. Epub 2022 Aug 18 doi: 10.1038/s41588-022-01104-0. PMID: 35982160Free PMC Article
Rodríguez-Palmero A, Boerrigter MM, Gómez-Andrés D, Aldinger KA, Marcos-Alcalde Í, Popp B, Everman DB, Lovgren AK, Arpin S, Bahrambeigi V, Beunders G, Bisgaard AM, Bjerregaard VA, Bruel AL, Challman TD, Cogné B, Coubes C, de Man SA, Denommé-Pichon AS, Dye TJ, Elmslie F, Feuk L, García-Miñaúr S, Gertler T, Giorgio E, Gruchy N, Haack TB, Haldeman-Englert CR, Haukanes BI, Hoyer J, Hurst ACE, Isidor B, Soller MJ, Kushary S, Kvarnung M, Landau YE, Leppig KA, Lindstrand A, Kleinendorst L, MacKenzie A, Mandrile G, Mendelsohn BA, Moghadasi S, Morton JE, Moutton S, Müller AJ, O'Leary M, Pacio-Míguez M, Palomares-Bralo M, Parikh S, Pfundt R, Pode-Shakked B, Rauch A, Repnikova E, Revah-Politi A, Ross MJ, Ruivenkamp CAL, Sarrazin E, Savatt JM, Schlüter A, Schönewolf-Greulich B, Shad Z, Shaw-Smith C, Shieh JT, Shohat M, Spranger S, Thiese H, Mau-Them FT, van Bon B, van de Burgt I, van de Laar IMBH, van Drie E, van Haelst MM, van Ravenswaaij-Arts CM, Verdura E, Vitobello A, Waldmüller S, Whiting S, Zweier C, Prada CE, de Vries BBA, Dobyns WB, Reiter SF, Gómez-Puertas P, Pujol A, Tümer Z
Genet Med 2021 May;23(5):888-899. Epub 2021 Feb 17 doi: 10.1038/s41436-020-01075-9. PMID: 33597769
Jaganathan K, Kyriazopoulou Panagiotopoulou S, McRae JF, Darbandi SF, Knowles D, Li YI, Kosmicki JA, Arbelaez J, Cui W, Schwartz GB, Chow ED, Kanterakis E, Gao H, Kia A, Batzoglou S, Sanders SJ, Farh KK
Cell 2019 Jan 24;176(3):535-548.e24. Epub 2019 Jan 17 doi: 10.1016/j.cell.2018.12.015. PMID: 30661751
Marucci S, Ragione LD, De Iaco G, Mococci T, Vicini M, Guastamacchia E, Triggiani V
Endocr Metab Immune Disord Drug Targets 2018;18(4):316-324. doi: 10.2174/1871530318666180213111637. PMID: 29437020

Recent systematic reviews

Vaquerizo-Serrano J, Salazar de Pablo G, Singh J, Santosh P
J Autism Dev Disord 2022 Apr;52(4):1568-1586. Epub 2021 May 15 doi: 10.1007/s10803-021-05046-0. PMID: 33993403Free PMC Article
Vaquerizo-Serrano J, Salazar De Pablo G, Singh J, Santosh P
Eur Psychiatry 2021 Dec 15;65(1):e4. doi: 10.1192/j.eurpsy.2021.2259. PMID: 34906264Free PMC Article
Ziats CA, Patterson WG, Friez M
Pediatr Neurol 2021 Jan;114:21-25. Epub 2020 Jun 28 doi: 10.1016/j.pediatrneurol.2020.06.011. PMID: 33189026
Kolevzon A, Delaby E, Berry-Kravis E, Buxbaum JD, Betancur C
Mol Autism 2019;10:50. Epub 2019 Dec 24 doi: 10.1186/s13229-019-0291-3. PMID: 31879555Free PMC Article
Griessenauer CJ, Farrell S, Sarkar A, Zand R, Abedi V, Holland N, Michael A, Cummings CL, Metpally R, Carey DJ, Goren O, Martin N, Hendrix P, Schirmer CM
J Cereb Blood Flow Metab 2018 Nov;38(11):1853-1871. Epub 2018 Sep 5 doi: 10.1177/0271678X18797958. PMID: 30182779Free PMC Article

Supplemental Content

Table of contents

    Clinical resources

    Practice guidelines

    • PubMed
      See practice and clinical guidelines in PubMed. The search results may include broader topics and may not capture all published guidelines. See the FAQ for details.
    • Bookshelf
      See practice and clinical guidelines in NCBI Bookshelf. The search results may include broader topics and may not capture all published guidelines. See the FAQ for details.

    Consumer resources

    Recent activity

    Your browsing activity is empty.

    Activity recording is turned off.

    Turn recording back on

    See more...