U.S. flag

An official website of the United States government

Format

Send to:

Choose Destination

Neurodevelopmental disorder with motor abnormalities, seizures, and facial dysmorphism(NEDMSF)

MedGen UID:
1052640
Concept ID:
CN376901
Disease or Syndrome
Synonyms: NEDMSF; PADDAS SYNDROME
 
Gene (location): PUM1 (1p35.2)
 
Monarch Initiative: MONDO:0958231
OMIM®: 620719

Definition

Neurodevelopmental disorder with motor abnormalities, seizures, and facial dysmorphism (NEDMSF) is characterized by global developmental delay, poor overall growth, early-onset seizures (in most patients), severely impaired motor development with hypotonia and/or ataxia, and dysmorphic facial features. Affected individuals have impaired intellectual development, which can be severe. Brain imaging may show thin corpus callosum, enlarged ventricles, or cerebellar atrophy (Gennarino et al., 2018; Voet et al., 2020). [from OMIM]

Professional guidelines

PubMed

Faundes V, Goh S, Akilapa R, Bezuidenhout H, Bjornsson HT, Bradley L, Brady AF, Brischoux-Boucher E, Brunner H, Bulk S, Canham N, Cody D, Dentici ML, Digilio MC, Elmslie F, Fry AE, Gill H, Hurst J, Johnson D, Julia S, Lachlan K, Lebel RR, Byler M, Gershon E, Lemire E, Gnazzo M, Lepri FR, Marchese A, McEntagart M, McGaughran J, Mizuno S, Okamoto N, Rieubland C, Rodgers J, Sasaki E, Scalais E, Scurr I, Suri M, van der Burgt I, Matsumoto N, Miyake N, Benoit V, Lederer D, Banka S
Genet Med 2021 Jul;23(7):1202-1210. Epub 2021 Mar 5 doi: 10.1038/s41436-021-01119-8. PMID: 33674768Free PMC Article
Levey EB, Stashinko E, Clegg NJ, Delgado MR
Am J Med Genet C Semin Med Genet 2010 Feb 15;154C(1):183-90. doi: 10.1002/ajmg.c.30254. PMID: 20104615
Rauch A, Schellmoser S, Kraus C, Dörr HG, Trautmann U, Altherr MR, Pfeiffer RA, Reis A
Am J Med Genet 2001 Apr 1;99(4):338-42. doi: 10.1002/ajmg.1203. PMID: 11252005

Recent clinical studies

Etiology

Smith AE, Jnah A, Newberry D
Neonatal Netw 2018 Sep;37(5):303-309. doi: 10.1891/0730-0832.37.5.303. PMID: 30567812
Levey EB, Stashinko E, Clegg NJ, Delgado MR
Am J Med Genet C Semin Med Genet 2010 Feb 15;154C(1):183-90. doi: 10.1002/ajmg.c.30254. PMID: 20104615
Lewis AJ, Simon EM, Barkovich AJ, Clegg NJ, Delgado MR, Levey E, Hahn JS
Neurology 2002 Dec 24;59(12):1860-5. doi: 10.1212/01.wnl.0000037483.31989.b9. PMID: 12499474
Rauch A, Schellmoser S, Kraus C, Dörr HG, Trautmann U, Altherr MR, Pfeiffer RA, Reis A
Am J Med Genet 2001 Apr 1;99(4):338-42. doi: 10.1002/ajmg.1203. PMID: 11252005
Gropman AL, Barkovich AJ, Vezina LG, Conry JA, Dubovsky EC, Packer RJ
Neuropediatrics 1997 Aug;28(4):198-203. doi: 10.1055/s-2007-973700. PMID: 9309709

Diagnosis

Bonardi CM, Bayat A, Madsen CG, Hammer TB, Reale C, Gardella E, Marjanovic D, Beniczky S, Møller RS, Rubboli G
Epileptic Disord 2022 Jun 1;24(3):577-582. doi: 10.1684/epd.2022.1423. PMID: 35770758
Cousin MA, Creighton BA, Breau KA, Spillmann RC, Torti E, Dontu S, Tripathi S, Ajit D, Edwards RJ, Afriyie S, Bay JC, Harper KM, Beltran AA, Munoz LJ, Falcon Rodriguez L, Stankewich MC, Person RE, Si Y, Normand EA, Blevins A, May AS, Bier L, Aggarwal V, Mancini GMS, van Slegtenhorst MA, Cremer K, Becker J, Engels H, Aretz S, MacKenzie JJ, Brilstra E, van Gassen KLI, van Jaarsveld RH, Oegema R, Parsons GM, Mark P, Helbig I, McKeown SE, Stratton R, Cogne B, Isidor B, Cacheiro P, Smedley D, Firth HV, Bierhals T, Kloth K, Weiss D, Fairley C, Shieh JT, Kritzer A, Jayakar P, Kurtz-Nelson E, Bernier RA, Wang T, Eichler EE, van de Laar IMBH, McConkie-Rosell A, McDonald MT, Kemppainen J, Lanpher BC, Schultz-Rogers LE, Gunderson LB, Pichurin PN, Yoon G, Zech M, Jech R, Winkelmann J; Undiagnosed Diseases Network; Genomics England Research Consortium, Beltran AS, Zimmermann MT, Temple B, Moy SS, Klee EW, Tan QK, Lorenzo DN
Nat Genet 2021 Jul;53(7):1006-1021. Epub 2021 Jul 1 doi: 10.1038/s41588-021-00886-z. PMID: 34211179Free PMC Article
Saredi S, Cauley ES, Ruggieri A, Spivey TM, Ardissone A, Mora M, Moroni I, Manzini MC
Muscle Nerve 2020 Aug;62(2):266-271. Epub 2020 May 27 doi: 10.1002/mus.26907. PMID: 32363625Free PMC Article
Schuurs-Hoeijmakers JH, Landsverk ML, Foulds N, Kukolich MK, Gavrilova RH, Greville-Heygate S, Hanson-Kahn A, Bernstein JA, Glass J, Chitayat D, Burrow TA, Husami A, Collins K, Wusik K, van der Aa N, Kooy F, Brown KT, Gadzicki D, Kini U, Alvarez S, Fernández-Jaén A, McGehee F, Selby K, Tarailo-Graovac M, Van Allen M, van Karnebeek CD, Stavropoulos DJ, Marshall CR, Merico D, Gregor A, Zweier C, Hopkin RJ, Chu YW, Chung BH, de Vries BB, Devriendt K, Hurles ME, Brunner HG; DDD study
Am J Med Genet A 2016 Mar;170(3):670-5. Epub 2016 Feb 3 doi: 10.1002/ajmg.a.37476. PMID: 26842493
Paprocka J, Jamroz E, Szwed-Białozyt B, Jezela-Stanek A, Kopyta I, Marszał E
Neurologist 2007 Sep;13(5):305-12. doi: 10.1097/01.nrl.0000253067.32759.aa. PMID: 17848870

Therapy

Da'as SI, Aamer W, Hasan W, Al-Maraghi A, Al-Kurbi A, Kilani H, AlRayahi J, Zamel K, Stotland MA, Fakhro KA
Cells 2020 Jul 27;9(8) doi: 10.3390/cells9081782. PMID: 32726939Free PMC Article
Schuurs-Hoeijmakers JH, Landsverk ML, Foulds N, Kukolich MK, Gavrilova RH, Greville-Heygate S, Hanson-Kahn A, Bernstein JA, Glass J, Chitayat D, Burrow TA, Husami A, Collins K, Wusik K, van der Aa N, Kooy F, Brown KT, Gadzicki D, Kini U, Alvarez S, Fernández-Jaén A, McGehee F, Selby K, Tarailo-Graovac M, Van Allen M, van Karnebeek CD, Stavropoulos DJ, Marshall CR, Merico D, Gregor A, Zweier C, Hopkin RJ, Chu YW, Chung BH, de Vries BB, Devriendt K, Hurles ME, Brunner HG; DDD study
Am J Med Genet A 2016 Mar;170(3):670-5. Epub 2016 Feb 3 doi: 10.1002/ajmg.a.37476. PMID: 26842493
Paprocka J, Jamroz E, Szwed-Białozyt B, Jezela-Stanek A, Kopyta I, Marszał E
Neurologist 2007 Sep;13(5):305-12. doi: 10.1097/01.nrl.0000253067.32759.aa. PMID: 17848870
Lewis AJ, Simon EM, Barkovich AJ, Clegg NJ, Delgado MR, Levey E, Hahn JS
Neurology 2002 Dec 24;59(12):1860-5. doi: 10.1212/01.wnl.0000037483.31989.b9. PMID: 12499474

Prognosis

Bonardi CM, Bayat A, Madsen CG, Hammer TB, Reale C, Gardella E, Marjanovic D, Beniczky S, Møller RS, Rubboli G
Epileptic Disord 2022 Jun 1;24(3):577-582. doi: 10.1684/epd.2022.1423. PMID: 35770758
Zorlu P, Eksioglu AS, Ozkan M, Tos T, Senel S
Genet Couns 2014;25(3):299-303. PMID: 25365852
Lewis AJ, Simon EM, Barkovich AJ, Clegg NJ, Delgado MR, Levey E, Hahn JS
Neurology 2002 Dec 24;59(12):1860-5. doi: 10.1212/01.wnl.0000037483.31989.b9. PMID: 12499474
Holmes GL, Logan WJ
Am J Dis Child 1980 Mar;134(3):262-6. doi: 10.1001/archpedi.1980.02130150020006. PMID: 7361733

Clinical prediction guides

Kaiyrzhanov R, Rad A, Lin SJ, Bertoli-Avella A, Kallemeijn WW, Godwin A, Zaki MS, Huang K, Lau T, Petree C, Efthymiou S, Karimiani EG, Hempel M, Normand EA, Rudnik-Schöneborn S, Schatz UA, Baggelaar MP, Ilyas M, Sultan T, Alvi JR, Ganieva M, Fowler B, Aanicai R, Tayfun GA, Al Saman A, Alswaid A, Amiri N, Asilova N, Shotelersuk V, Yeetong P, Azam M, Babaei M, Monajemi GB, Mohammadi P, Samie S, Banu SH, Pinto Basto J, Kortüm F, Bauer M, Bauer P, Beetz C, Garshasbi M, Issa AH, Eyaid W, Ahmed H, Hashemi N, Hassanpour K, Herman I, Ibrohimov S, Abdul-Majeed BA, Imdad M, Isrofilov M, Kaiyal Q, Khan S, Kirmse B, Koster J, Lourenço CM, Mitani T, Moldovan O, Murphy D, Najafi M, Pehlivan D, Rocha ME, Salpietro V, Schmidts M, Shalata A, Mahroum M, Talbeya JK, Taylor RW, Vazquez D, Vetro A, Waterham HR, Zaman M, Schrader TA, Chung WK, Guerrini R, Lupski JR, Gleeson J, Suri M, Jamshidi Y, Bhatia KP, Vona B, Schrader M, Severino M, Guille M, Tate EW, Varshney GK, Houlden H, Maroofian R
Brain 2024 Apr 4;147(4):1436-1456. doi: 10.1093/brain/awad380. PMID: 37951597Free PMC Article
Varela T, Varela D, Martins G, Conceição N, Cancela ML
Sci Rep 2022 Jun 4;12(1):9325. doi: 10.1038/s41598-022-13364-1. PMID: 35665761Free PMC Article
Donnarumma B, Riccio MP, Terrone G, Palma M, Strisciuglio P, Scala I
Ital J Pediatr 2021 Jul 2;47(1):148. doi: 10.1186/s13052-021-01101-9. PMID: 34215294Free PMC Article
Cousin MA, Creighton BA, Breau KA, Spillmann RC, Torti E, Dontu S, Tripathi S, Ajit D, Edwards RJ, Afriyie S, Bay JC, Harper KM, Beltran AA, Munoz LJ, Falcon Rodriguez L, Stankewich MC, Person RE, Si Y, Normand EA, Blevins A, May AS, Bier L, Aggarwal V, Mancini GMS, van Slegtenhorst MA, Cremer K, Becker J, Engels H, Aretz S, MacKenzie JJ, Brilstra E, van Gassen KLI, van Jaarsveld RH, Oegema R, Parsons GM, Mark P, Helbig I, McKeown SE, Stratton R, Cogne B, Isidor B, Cacheiro P, Smedley D, Firth HV, Bierhals T, Kloth K, Weiss D, Fairley C, Shieh JT, Kritzer A, Jayakar P, Kurtz-Nelson E, Bernier RA, Wang T, Eichler EE, van de Laar IMBH, McConkie-Rosell A, McDonald MT, Kemppainen J, Lanpher BC, Schultz-Rogers LE, Gunderson LB, Pichurin PN, Yoon G, Zech M, Jech R, Winkelmann J; Undiagnosed Diseases Network; Genomics England Research Consortium, Beltran AS, Zimmermann MT, Temple B, Moy SS, Klee EW, Tan QK, Lorenzo DN
Nat Genet 2021 Jul;53(7):1006-1021. Epub 2021 Jul 1 doi: 10.1038/s41588-021-00886-z. PMID: 34211179Free PMC Article
Levey EB, Stashinko E, Clegg NJ, Delgado MR
Am J Med Genet C Semin Med Genet 2010 Feb 15;154C(1):183-90. doi: 10.1002/ajmg.c.30254. PMID: 20104615

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...