Literature DB >> 23798012

Newborn screening for lysosomal storage disorders and other neuronopathic conditions.

Dietrich Matern1, Devin Oglesbee, Silvia Tortorelli.   

Abstract

Newborn screening (NBS) is a public health program aimed at identifying treatable conditions in presymptomatic newborns to avoid premature mortality, morbidity, and disabilities. Currently, every newborn in the Unites States is screened for at least 29 conditions where evidence suggests that early detection is possible and beneficial. With new or improved treatment options and development of high-throughput screening tests, additional conditions have been proposed for inclusion into NBS programs. Among those are several conditions with a strong neuronopathic component. Some of these conditions have already been added to a few national and international screening programs, whereas others are undergoing pilot studies to determine the test performance metrics. Here, we review the current state of NBS for 13 lysosomal storage disorders, X-adrenoleukodystrophy, Wilson disease, and Friedreich ataxia.
Copyright © 2013 Wiley Periodicals, Inc., a Wiley company.

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Year:  2013        PMID: 23798012      PMCID: PMC3902890          DOI: 10.1002/ddrr.1117

Source DB:  PubMed          Journal:  Dev Disabil Res Rev        ISSN: 1940-5529


  57 in total

Review 1.  Recent developments in therapeutic approaches for lysosomal storage diseases.

Authors:  Lorena Urbanelli; Alessandro Magini; Alice Polchi; Mario Polidoro; Carla Emiliani
Journal:  Recent Pat CNS Drug Discov       Date:  2011-01

Review 2.  Friedreich's ataxia: pathology, pathogenesis, and molecular genetics.

Authors:  Arnulf H Koeppen
Journal:  J Neurol Sci       Date:  2011-04-15       Impact factor: 3.181

3.  Mutations in SLC33A1 cause a lethal autosomal-recessive disorder with congenital cataracts, hearing loss, and low serum copper and ceruloplasmin.

Authors:  Peter Huppke; Cornelia Brendel; Vera Kalscheuer; Georg Christoph Korenke; Iris Marquardt; Peter Freisinger; John Christodoulou; Merle Hillebrand; Gaele Pitelet; Callum Wilson; Ursula Gruber-Sedlmayr; Reinhard Ullmann; Stefan Haas; Orly Elpeleg; Gudrun Nürnberg; Peter Nürnberg; Shzeena Dad; Lisbeth Birk Møller; Stephen G Kaler; Jutta Gärtner
Journal:  Am J Hum Genet       Date:  2012-01-13       Impact factor: 11.025

4.  Screening patients referred to a metabolic clinic for lysosomal storage disorders.

Authors:  Maria Fuller; Justin N Tucker; Debbie L Lang; Caroline J Dean; Michael J Fietz; Peter J Meikle; John J Hopwood
Journal:  J Med Genet       Date:  2011-03-17       Impact factor: 6.318

5.  Outcomes after allogeneic hematopoietic cell transplantation for childhood cerebral adrenoleukodystrophy: the largest single-institution cohort report.

Authors:  Weston P Miller; Steven M Rothman; David Nascene; Teresa Kivisto; Todd E DeFor; Richard S Ziegler; Julie Eisengart; Kara Leiser; Gerald Raymond; Troy C Lund; Jakub Tolar; Paul J Orchard
Journal:  Blood       Date:  2011-05-17       Impact factor: 22.113

6.  Comparative triplex tandem mass spectrometry assays of lysosomal enzyme activities in dried blood spots using fast liquid chromatography: application to newborn screening of Pompe, Fabry, and Hurler diseases.

Authors:  Zdeněk Spáčil; Susan Elliott; Steven L Reeber; Michael H Gelb; C Ronald Scott; František Tureček
Journal:  Anal Chem       Date:  2011-05-17       Impact factor: 6.986

7.  Digital microfluidic platform for multiplexing enzyme assays: implications for lysosomal storage disease screening in newborns.

Authors:  Ramakrishna S Sista; Allen E Eckhardt; Tong Wang; Carrie Graham; Jeremy L Rouse; Scott M Norton; Vijay Srinivasan; Michael G Pollack; Adviye A Tolun; Deeksha Bali; David S Millington; Vamsee K Pamula
Journal:  Clin Chem       Date:  2011-08-22       Impact factor: 8.327

8.  Clinical validation of cutoff target ranges in newborn screening of metabolic disorders by tandem mass spectrometry: a worldwide collaborative project.

Authors:  David M S McHugh; Cynthia A Cameron; Jose E Abdenur; Mahera Abdulrahman; Ona Adair; Shahira Ahmed Al Nuaimi; Henrik Åhlman; Jennifer J Allen; Italo Antonozzi; Shaina Archer; Sylvia Au; Christiane Auray-Blais; Mei Baker; Fiona Bamforth; Kinga Beckmann; Gessi Bentz Pino; Stanton L Berberich; Robert Binard; François Boemer; Jim Bonham; Nancy N Breen; Sandra C Bryant; Michele Caggana; S Graham Caldwell; Marta Camilot; Carlene Campbell; Claudia Carducci; Sandra C Bryant; Michele Caggana; S Graham Caldwell; Marta Camilot; Carlene Campbell; Claudia Carducci; Rohit Cariappa; Clover Carlisle; Ubaldo Caruso; Michela Cassanello; Ane Miren Castilla; Daisy E Castiñeiras Ramos; Pranesh Chakraborty; Ram Chandrasekar; Alfredo Chardon Ramos; David Cheillan; Yin-Hsiu Chien; Thomas A Childs; Petr Chrastina; Yuri Cleverthon Sica; Jose Angel Cocho de Juan; Maria Elena Colandre; Veronica Cornejo Espinoza; Gaetano Corso; Robert Currier; Denis Cyr; Noemi Czuczy; Oceania D'Apolito; Tim Davis; Monique G de Sain-Van der Velden; Carmen Delgado Pecellin; Iole Maria Di Gangi; Cristina Maria Di Stefano; Yannis Dotsikas; Melanie Downing; Stephen M Downs; Bonifacio Dy; Mark Dymerski; Inmaculada Rueda; Bert Elvers; Roger Eaton; Barbara M Eckerd; Fatma El Mougy; Sarah Eroh; Mercedes Espada; Catherine Evans; Sandy Fawbush; Kristel F Fijolek; Lawrence Fisher; Leifur Franzson; Dianne M Frazier; Luciana R C Garcia; Maria Sierra García-Valdecasas Bermejo; Dimitar Gavrilov; Rosemarie Gerace; Giuseppe Giordano; Yolanda González Irazabal; Lawrence C Greed; Robert Grier; Elyse Grycki; Xuefan Gu; Fizza Gulamali-Majid; Arthur F Hagar; Lianshu Han; W Harry Hannon; Christa Haslip; Fayza Abdelhamid Hassan; Miao He; Amy Hietala; Leslie Himstedt; Gary L Hoffman; William Hoffman; Philis Hoggatt; Patrick V Hopkins; David M Hougaard; Kerie Hughes; Patricia R Hunt; Wuh-Liang Hwu; June Hynes; Isabel Ibarra-González; Cindy A Ingham; Maria Ivanova; Ward B Jacox; Catharine John; John P Johnson; Jón J Jónsson; Eszter Karg; David Kasper; Brenda Klopper; Dimitris Katakouzinos; Issam Khneisser; Detlef Knoll; Hirinori Kobayashi; Ronald Koneski; Viktor Kozich; Rasoul Kouapei; Dirk Kohlmueller; Ivo Kremensky; Giancarlo la Marca; Marcia Lavochkin; Soo-Youn Lee; Denis C Lehotay; Aida Lemes; Joyce Lepage; Barbara Lesko; Barry Lewis; Carol Lim; Sharon Linard; Martin Lindner; Michele A Lloyd-Puryear; Fred Lorey; Yannis L Loukas; Julie Luedtke; Neil Maffitt; J Fergall Magee; Adrienne Manning; Shawn Manos; Sandrine Marie; Sônia Marchezi Hadachi; Gregg Marquardt; Stephen J Martin; Dietrich Matern; Stephanie K Mayfield Gibson; Philip Mayne; Tonya D McCallister; Mark McCann; Julie McClure; James J McGill; Christine D McKeever; Barbara McNeilly; Mark A Morrissey; Paraskevi Moutsatsou; Eleanor A Mulcahy; Dimitris Nikoloudis; Bent Norgaard-Pedersen; Devin Oglesbee; Mariusz Oltarzewski; Daniela Ombrone; Jelili Ojodu; Vagelis Papakonstantinou; Sherly Pardo Reoyo; Hyung-Doo Park; Marzia Pasquali; Elisabetta Pasquini; Pallavi Patel; Kenneth A Pass; Colleen Peterson; Rolf D Pettersen; James J Pitt; Sherry Poh; Arnold Pollak; Cory Porter; Philip A Poston; Ricky W Price; Cecilia Queijo; Jonessy Quesada; Edward Randell; Enzo Ranieri; Kimiyo Raymond; John E Reddic; Alejandra Reuben; Charla Ricciardi; Piero Rinaldo; Jeff D Rivera; Alicia Roberts; Hugo Rocha; Geraldine Roche; Cheryl Rochman Greenberg; José María Egea Mellado; María Jesús Juan-Fita; Consuelo Ruiz; Margherita Ruoppolo; S Lane Rutledge; Euijung Ryu; Christine Saban; Inderneel Sahai; Maria Isabel Salazar García-Blanco; Pedro Santiago-Borrero; Andrea Schenone; Roland Schoos; Barb Schweitzer; Patricia Scott; Margretta R Seashore; Mary A Seeterlin; David E Sesser; Darrin W Sevier; Scott M Shone; Graham Sinclair; Victor A Skrinska; Eleanor L Stanley; Erin T Strovel; April L Studinski Jones; Sherlykutty Sunny; Zoltan Takats; Tijen Tanyalcin; Francesca Teofoli; J Robert Thompson; Kathy Tomashitis; Mouseline Torquado Domingos; Jasmin Torres; Rosario Torres; Silvia Tortorelli; Sandor Turi; Kimberley Turner; Nick Tzanakos; Alf G Valiente; Hillary Vallance; Marcela Vela-Amieva; Laura Vilarinho; Ulrika von Döbeln; Marie-Francoise Vincent; B Chris Vorster; Michael S Watson; Dianne Webster; Sheila Weiss; Bridget Wilcken; Veronica Wiley; Sharon K Williams; Sharon A Willis; Michael Woontner; Katherine Wright; Raquel Yahyaoui; Seiji Yamaguchi; Melissa Yssel; Wendy M Zakowicz
Journal:  Genet Med       Date:  2011-03       Impact factor: 8.822

9.  The impact of false-positive newborn screening results on families: a qualitative study.

Authors:  Johanna L Schmidt; Karen Castellanos-Brown; Saltanat Childress; Natasha Bonhomme; Julianne S Oktay; Sharon F Terry; Penny Kyler; Amy Davidoff; Carol Greene
Journal:  Genet Med       Date:  2012-01       Impact factor: 8.822

10.  Lysosomal storage diseases: diagnostic confirmation and management of presymptomatic individuals.

Authors:  Raymond Y Wang; Olaf A Bodamer; Michael S Watson; William R Wilcox
Journal:  Genet Med       Date:  2011-05       Impact factor: 8.822

View more
  8 in total

1.  Newborn Screening for Lysosomal Storage Disorders: Views of Genetic Healthcare Providers.

Authors:  Emily C Lisi; Shawn E McCandless
Journal:  J Genet Couns       Date:  2015-08-29       Impact factor: 2.537

2.  Effect of temperature on lysosomal enzyme activity during preparation and storage of dried blood spots.

Authors:  Manjunath Supriya; Tanima De; Rita Christopher
Journal:  J Clin Lab Anal       Date:  2017-03-27       Impact factor: 2.352

Review 3.  Lysosomal enzyme replacement therapies: Historical development, clinical outcomes, and future perspectives.

Authors:  Melani Solomon; Silvia Muro
Journal:  Adv Drug Deliv Rev       Date:  2017-05-11       Impact factor: 15.470

Review 4.  Differential diagnosis of Mendelian and mitochondrial disorders in patients with suspected multiple sclerosis.

Authors:  James D Weisfeld-Adams; Ilana B Katz Sand; Justin M Honce; Fred D Lublin
Journal:  Brain       Date:  2015-01-29       Impact factor: 13.501

Review 5.  Newborn screening for lysosomal storage diseases.

Authors:  Michael H Gelb; C Ronald Scott; Frantisek Turecek
Journal:  Clin Chem       Date:  2014-12-04       Impact factor: 8.327

6.  Enzymatic Screening and Diagnosis of Lysosomal Storage Diseases.

Authors:  Chunli Yu; Qin Sun; Hui Zhou
Journal:  N Am J Med Sci (Boston)       Date:  2013

Review 7.  The genetic landscape of X-linked adrenoleukodystrophy: inheritance, mutations, modifier genes, and diagnosis.

Authors:  Christoph Wiesinger; Florian S Eichler; Johannes Berger
Journal:  Appl Clin Genet       Date:  2015-05-02

8.  Case reports of metabolic disorders from Nepal.

Authors:  Arti Sharma Pandey
Journal:  Mol Genet Metab Rep       Date:  2019-11-19
  8 in total

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