Literature DB >> 28814539

FORWARD: A Registry and Longitudinal Clinical Database to Study Fragile X Syndrome.

Stephanie L Sherman1, Sharon A Kidd2, Catharine Riley3, Elizabeth Berry-Kravis4,5,6, Howard F Andrews7, Robert M Miller2, Sharyn Lincoln8, Mark Swanson3, Walter E Kaufmann9,10, W Ted Brown11.   

Abstract

BACKGROUND AND
OBJECTIVE: Advances in the care of patients with fragile X syndrome (FXS) have been hampered by lack of data. This deficiency has produced fragmentary knowledge regarding the natural history of this condition, healthcare needs, and the effects of the disease on caregivers. To remedy this deficiency, the Fragile X Clinic and Research Consortium was established to facilitate research. Through a collective effort, the Fragile X Clinic and Research Consortium developed the Fragile X Online Registry With Accessible Research Database (FORWARD) to facilitate multisite data collection. This report describes FORWARD and the way it can be used to improve health and quality of life of FXS patients and their relatives and caregivers.
METHODS: FORWARD collects demographic information on individuals with FXS and their family members (affected and unaffected) through a 1-time registry form. The longitudinal database collects clinician- and parent-reported data on individuals diagnosed with FXS, focused on those who are 0 to 24 years of age, although individuals of any age can participate.
RESULTS: The registry includes >2300 registrants (data collected September 7, 2009 to August 31, 2014). The longitudinal database includes data on 713 individuals diagnosed with FXS (data collected September 7, 2012 to August 31, 2014). Longitudinal data continue to be collected on enrolled patients along with baseline data on new patients.
CONCLUSIONS: FORWARD represents the largest resource of clinical and demographic data for the FXS population in the United States. These data can be used to advance our understanding of FXS: the impact of cooccurring conditions, the impact on the day-to-day lives of individuals living with FXS and their families, and short-term and long-term outcomes.
Copyright © 2017 by the American Academy of Pediatrics.

Entities:  

Mesh:

Year:  2017        PMID: 28814539      PMCID: PMC5621599          DOI: 10.1542/peds.2016-1159E

Source DB:  PubMed          Journal:  Pediatrics        ISSN: 0031-4005            Impact factor:   7.124


  41 in total

1.  Fragile X genotype characterized by an unstable region of DNA.

Authors:  S Yu; M Pritchard; E Kremer; M Lynch; J Nancarrow; E Baker; K Holman; J C Mulley; S T Warren; D Schlessinger
Journal:  Science       Date:  1991-05-24       Impact factor: 47.728

2.  FMR1 fully expanded mutation with minimal methylation in a high functioning fragile X male.

Authors:  Z Wang; A K Taylor; J A Bridge
Journal:  J Med Genet       Date:  1996-05       Impact factor: 6.318

3.  Cognitive, language and social-cognitive skills of individuals with fragile X syndrome with and without autism.

Authors:  P Lewis; L Abbeduto; M Murphy; E Richmond; N Giles; L Bruno; S Schroeder
Journal:  J Intellect Disabil Res       Date:  2006-07

4.  Mosaicism for a full mutation, premutation, and deletion of the CGG repeats results in 22% FMRP and elevated FMR1 mRNA levels in a high-functioning fragile X male.

Authors:  Xiao-Dong Han; Berkley R Powell; Judith L Phalin; Farid F Chehab
Journal:  Am J Med Genet A       Date:  2006-07-01       Impact factor: 2.802

5.  Prevalence, nature, and correlates of sleep problems among children with fragile X syndrome based on a large scale parent survey.

Authors:  Rebecca Kronk; Ellen E Bishop; Melissa Raspa; Julie O Bickel; Daniel A Mandel; Donald B Bailey
Journal:  Sleep       Date:  2010-05       Impact factor: 5.849

6.  Co-occurring conditions associated with FMR1 gene variations: findings from a national parent survey.

Authors:  Donald B Bailey; Melissa Raspa; Murrey Olmsted; David B Holiday
Journal:  Am J Med Genet A       Date:  2008-08-15       Impact factor: 2.802

7.  Normal phenotype in two brothers with a full FMR1 mutation.

Authors:  H J Smeets; A P Smits; C E Verheij; J P Theelen; R Willemsen; I van de Burgt; A T Hoogeveen; J C Oosterwijk; B A Oostra
Journal:  Hum Mol Genet       Date:  1995-11       Impact factor: 6.150

8.  Autism profiles of males with fragile X syndrome.

Authors:  Susan W Harris; David Hessl; Beth Goodlin-Jones; Jessica Ferranti; Susan Bacalman; Ingrid Barbato; Flora Tassone; Paul J Hagerman; Herman Herman; Randi J Hagerman
Journal:  Am J Ment Retard       Date:  2008-11

9.  Prevalence of carriers of premutation-size alleles of the FMRI gene--and implications for the population genetics of the fragile X syndrome.

Authors:  F Rousseau; P Rouillard; M L Morel; E W Khandjian; K Morgan
Journal:  Am J Hum Genet       Date:  1995-11       Impact factor: 11.025

10.  EXPLAIN Fragile-X: an explorative, longitudinal study on the characterization, treatment pathways, and patient-related outcomes of Fragile X Syndrome.

Authors:  Frank Haessler; Franziska Gaese; Michael Colla; Michael Huss; Christoph Kretschmar; Marc Brinkman; Heike Schieb; Helmut Peters; Samuel Elstner; David Pittrow
Journal:  BMC Psychiatry       Date:  2013-12-19       Impact factor: 3.630

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  12 in total

1.  Preventive care services and health behaviors in children with fragile X syndrome.

Authors:  Kendra E Gilbertson; Hannah L Jackson; Eric J Dziuban; Stephanie L Sherman; Elizabeth M Berry-Kravis; Craig A Erickson; Rodolfo Valdez
Journal:  Disabil Health J       Date:  2019-05-03       Impact factor: 2.554

2.  Toilet Training in Fragile X Syndrome.

Authors:  Elizabeth Berry-Kravis; Sharon A Kidd; Ave M Lachiewicz; Tse Hwei Choo; Nicole Tartaglia; Devadrita Talapatra; Christina Aguirre-Kolb; Howard Andrews; Karen Riley
Journal:  J Dev Behav Pediatr       Date:  2019-12       Impact factor: 2.225

3.  Pharmacologic Interventions for Irritability, Aggression, Agitation and Self-Injurious Behavior in Fragile X Syndrome: An Initial Cross-Sectional Analysis.

Authors:  Eleanor M Eckert; Kelli C Dominick; Ernest V Pedapati; Logan K Wink; Rebecca C Shaffer; Howard Andrews; Tse-Hwei Choo; Chen Chen; Walter E Kaufmann; Nicole Tartaglia; Elizabeth M Berry-Kravis; Craig A Erickson
Journal:  J Autism Dev Disord       Date:  2019-11

Review 4.  Developmental studies in fragile X syndrome.

Authors:  Khaleel A Razak; Kelli C Dominick; Craig A Erickson
Journal:  J Neurodev Disord       Date:  2020-05-02       Impact factor: 4.025

5.  Delineating Repetitive Behavior Profiles across the Lifespan in Fragile X Syndrome.

Authors:  Debra L Reisinger; Rebecca C Shaffer; Nicole Tartaglia; Elizabeth Berry-Kravis; Craig A Erickson
Journal:  Brain Sci       Date:  2020-04-17

6.  Soy-Based Infant Formula is Associated with an Increased Prevalence of Comorbidities in Fragile X Syndrome.

Authors:  Cara J Westmark; Chad Kniss; Emmanuel Sampene; Angel Wang; Amie Milunovich; Kelly Elver; David Hessl; Amy Talboy; Jonathon Picker; Barbara Haas-Givler; Amy Esler; Andrea L Gropman; Ryan Uy; Craig Erickson; Milen Velinov; Nicole Tartaglia; Elizabeth M Berry-Kravis
Journal:  Nutrients       Date:  2020-10-14       Impact factor: 5.717

7.  Seizures in Fragile X Syndrome: Associations and Longitudinal Analysis of a Large Clinic-Based Cohort.

Authors:  Elizabeth Berry-Kravis; Robyn A Filipink; Richard E Frye; Sailaja Golla; Stephanie M Morris; Howard Andrews; Tse-Hwei Choo; Walter E Kaufmann
Journal:  Front Pediatr       Date:  2021-12-30       Impact factor: 3.418

8.  Consumption of Breast Milk Is Associated with Decreased Prevalence of Autism in Fragile X Syndrome.

Authors:  Cara J Westmark
Journal:  Nutrients       Date:  2021-05-24       Impact factor: 5.717

9.  Evaluating Social Interactions Using the Autism Screening Instrument for Education Planning-3rd Edition (ASIEP-3): Interaction Assessment in Children and Adults with Fragile X Syndrome.

Authors:  Lisa Cordeiro; Marcia Braden; Elizabeth Coan; Nanastasia Welnick; Tanea Tanda; Nicole Tartaglia
Journal:  Brain Sci       Date:  2020-04-22

10.  Adaptive Skills in FXS: A Review of the Literature and Evaluation of the PEDI-Computer Adaptive Test (PEDI-CAT) to Measure Adaptive Skills.

Authors:  Lisa Cordeiro; Adrienne Villagomez; Deanna Swain; Sophia Deklotz; Nicole Tartaglia
Journal:  Brain Sci       Date:  2020-06-06
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