Literature DB >> 31074943

Updating the neurodevelopmental profile of Alazami syndrome: Illustrating the role of developmental assessment in rare genetic disorders.

Monica H Wojcik1,2, Kate Linnea3, Joan M Stoler1, Leonard Rappaport3.   

Abstract

Alazami syndrome, caused by biallelic pathogenic variants in LARP7, is a recently-described rare genetic disorder, with 17 patients currently reported in the literature. We present a case of a male infant referred for genetics evaluation at 5 months of age, found at 17 months of age to have Alazami syndrome. He was promptly referred for developmental evaluation, where he was found to be higher functioning than prior reports of individuals with this condition. This demonstrates the neurodevelopmental phenotypic variability seen in rare genetic disorders; it also demonstrates the important role of developmental programs to measure and track outcomes and provide support for infants with genetic disorders that put them at risk of developmental disabilities.
© 2019 Wiley Periodicals, Inc.

Entities:  

Keywords:  zzm321990LARP7; Alazami syndrome; developmental disabilities; primordial dwarfism

Mesh:

Substances:

Year:  2019        PMID: 31074943      PMCID: PMC6663627          DOI: 10.1002/ajmg.a.61189

Source DB:  PubMed          Journal:  Am J Med Genet A        ISSN: 1552-4825            Impact factor:   2.802


  10 in total

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Journal:  Nature       Date:  2011-09-21       Impact factor: 49.962

2.  Compound heterozygous variants in the LARP7 gene as a cause of Alazami syndrome in a Caucasian female with significant failure to thrive, short stature, and developmental disability.

Authors:  Tina T Ling; Susanna Sorrentino
Journal:  Am J Med Genet A       Date:  2015-09-16       Impact factor: 2.802

3.  Parents' perceptions of functional abilities in people with Down syndrome.

Authors:  Gert de Graaf; Susan P Levine; Richard Goldstein; Brian G Skotko
Journal:  Am J Med Genet A       Date:  2018-12-24       Impact factor: 2.802

4.  Loss of function mutation in LARP7, chaperone of 7SK ncRNA, causes a syndrome of facial dysmorphism, intellectual disability, and primordial dwarfism.

Authors:  Anas M Alazami; Mohammad Al-Owain; Fatema Alzahrani; Taghreed Shuaib; Hussain Al-Shamrani; Yahya H Al-Falki; Saleh M Al-Qahtani; Tarfa Alsheddi; Dilek Colak; Fowzan S Alkuraya
Journal:  Hum Mutat       Date:  2012-08-30       Impact factor: 4.878

5.  Effectiveness of exome and genome sequencing guided by acuity of illness for diagnosis of neurodevelopmental disorders.

Authors:  Sarah E Soden; Carol J Saunders; Laurel K Willig; Emily G Farrow; Laurie D Smith; Josh E Petrikin; Jean-Baptiste LePichon; Neil A Miller; Isabelle Thiffault; Darrell L Dinwiddie; Greyson Twist; Aaron Noll; Bryce A Heese; Lee Zellmer; Andrea M Atherton; Ahmed T Abdelmoity; Nicole Safina; Sarah S Nyp; Britton Zuccarelli; Ingrid A Larson; Ann Modrcin; Suzanne Herd; Mitchell Creed; Zhaohui Ye; Xuan Yuan; Robert A Brodsky; Stephen F Kingsmore
Journal:  Sci Transl Med       Date:  2014-12-03       Impact factor: 17.956

6.  LARP7 variants and further delineation of the Alazami syndrome phenotypic spectrum among primordial dwarfisms: 2 sisters.

Authors:  Marion Imbert-Bouteille; Frédéric Tran Mau Them; Julien Thevenon; Thomas Guignard; Vincent Gatinois; Jean-Baptiste Riviere; Anne Boland; Vincent Meyer; Jean-François Deleuze; Elodie Sanchez; Florence Apparailly; David Geneviève; Marjolaine Willems
Journal:  Eur J Med Genet       Date:  2018-07-10       Impact factor: 2.708

7.  Broadening the phenotypic spectrum of pathogenic LARP7 variants: two cases with intellectual disability, variable growth retardation and distinct facial features.

Authors:  Iris H I M Hollink; Majid Alfadhel; Anwar S Al-Wakeel; Farough Ababneh; Rolph Pfundt; Stella A de Man; Rami Abou Jamra; Arndt Rolfs; Aida M Bertoli-Avella; Ingrid M B H van de Laar
Journal:  J Hum Genet       Date:  2015-11-26       Impact factor: 3.172

8.  Genetic diagnosis of developmental disorders in the DDD study: a scalable analysis of genome-wide research data.

Authors:  Caroline F Wright; Tomas W Fitzgerald; Wendy D Jones; Stephen Clayton; Jeremy F McRae; Margriet van Kogelenberg; Daniel A King; Kirsty Ambridge; Daniel M Barrett; Tanya Bayzetinova; A Paul Bevan; Eugene Bragin; Eleni A Chatzimichali; Susan Gribble; Philip Jones; Netravathi Krishnappa; Laura E Mason; Ray Miller; Katherine I Morley; Vijaya Parthiban; Elena Prigmore; Diana Rajan; Alejandro Sifrim; G Jawahar Swaminathan; Adrian R Tivey; Anna Middleton; Michael Parker; Nigel P Carter; Jeffrey C Barrett; Matthew E Hurles; David R FitzPatrick; Helen V Firth
Journal:  Lancet       Date:  2014-12-17       Impact factor: 79.321

9.  Impaired telomere maintenance in Alazami syndrome patients with LARP7 deficiency.

Authors:  Brody Holohan; Wanil Kim; Tsung-Po Lai; Hirotoshi Hoshiyama; Ning Zhang; Anas M Alazami; Woodring E Wright; M Stephen Meyn; Fowzan S Alkuraya; Jerry W Shay
Journal:  BMC Genomics       Date:  2016-10-17       Impact factor: 3.969

10.  Novel compound heterozygous variants in the LARP7 gene in a patient with Alazami syndrome.

Authors:  Sumito Dateki; Tasuku Kitajima; Toshiharu Kihara; Satoshi Watanabe; Koh-Ichiro Yoshiura; Hiroyuki Moriuchi
Journal:  Hum Genome Var       Date:  2018-03-29
  10 in total
  5 in total

Review 1.  Developmental Support for Infants With Genetic Disorders.

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Journal:  Pediatrics       Date:  2020-05       Impact factor: 7.124

2.  Stabilize and connect: the role of LARP7 in nuclear non-coding RNA metabolism.

Authors:  Daniele Hasler; Gunter Meister; Utz Fischer
Journal:  RNA Biol       Date:  2020-06-03       Impact factor: 4.652

3.  Alazami syndrome: the first case of papillary thyroid carcinoma.

Authors:  Ivan Ivanovski; Stefano Giuseppe Caraffi; Elisa Magnani; Simonetta Rosato; Marzia Pollazzon; Leslie Matalonga; Simonetta Piana; Davide Nicoli; Chiara Baldo; Sergio Bernasconi; Andrea Frasoldati; Orsetta Zuffardi; Livia Garavelli
Journal:  J Hum Genet       Date:  2019-10-28       Impact factor: 3.172

4.  Compound Phenotype Due to Recessive Variants in LARP7 and OTOG Genes Disclosed by an Integrated Approach of SNP-Array and Whole Exome Sequencing.

Authors:  Pietro Palumbo; Orazio Palumbo; Maria Pia Leone; Ester di Muro; Stefano Castellana; Luigi Bisceglia; Tommaso Mazza; Massimo Carella; Marco Castori
Journal:  Genes (Basel)       Date:  2020-03-31       Impact factor: 4.096

5.  Patient with Phenylketonuria and Intellectual Disability-Problem Not Always Caused Exclusively by Insufficient Metabolic Control (Coexistence of PKU and Alazami Syndrome).

Authors:  Michał Patalan; Alicja Leśniak; Krzysztof Bernatowicz; Hanna Romanowska; Elżbieta Krzywińska-Zdeb; Mieczysław Walczak; Maria Giżewska
Journal:  Int J Environ Res Public Health       Date:  2022-02-24       Impact factor: 3.390

  5 in total

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