Literature DB >> 25122204

Population-based incidence and prevalence of facioscapulohumeral dystrophy.

Johanna C W Deenen1, Hisse Arnts2, Silvère M van der Maarel2, George W Padberg2, Jan J G M Verschuuren2, Egbert Bakker2, Stephanie S Weinreich2, André L M Verbeek2, Baziel G M van Engelen2.   

Abstract

OBJECTIVE: To determine the incidence and prevalence of facioscapulohumeral muscular dystrophy (FSHD) in the Netherlands.
METHODS: Using 3-source capture-recapture methodology, we estimated the total yearly number of newly found symptomatic individuals with FSHD, including those not registered in any of the 3 sources. To this end, symptomatic individuals with FSHD were available from 3 large population-based registries in the Netherlands if diagnosed within a 10-year period (January 1, 2001 to December 31, 2010). Multiplication of the incidence and disease duration delivered the prevalence estimate.
RESULTS: On average, 52 people are newly diagnosed with FSHD every year. This results in an incidence rate of 0.3/100,000 person-years in the Netherlands. The prevalence rate was 12/100,000, equivalent to 2,000 affected individuals.
CONCLUSIONS: We present population-based incidence and prevalence estimates regarding symptomatic individuals with FSHD, including an estimation of the number of symptomatic individuals not present in any of the 3 used registries. This study shows that the total number of symptomatic persons with FSHD in the population may well be underestimated and a considerable number of affected individuals remain undiagnosed. This suggests that FSHD is one of the most prevalent neuromuscular disorders.
© 2014 American Academy of Neurology.

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Year:  2014        PMID: 25122204      PMCID: PMC4166358          DOI: 10.1212/WNL.0000000000000797

Source DB:  PubMed          Journal:  Neurology        ISSN: 0028-3878            Impact factor:   9.910


  6 in total

1.  Chromosome 4q DNA rearrangements associated with facioscapulohumeral muscular dystrophy.

Authors:  C Wijmenga; J E Hewitt; L A Sandkuijl; L N Clark; T J Wright; H G Dauwerse; A M Gruter; M H Hofker; P Moerer; R Williamson
Journal:  Nat Genet       Date:  1992-09       Impact factor: 38.330

2.  Descriptive epidemiology of selected neurologic and myopathic disorders with particular reference to a survey in Rochester, Minnesota.

Authors:  L T KURLAND
Journal:  J Chronic Dis       Date:  1958-10

3.  A unifying genetic model for facioscapulohumeral muscular dystrophy.

Authors:  Richard J L F Lemmers; Patrick J van der Vliet; Rinse Klooster; Sabrina Sacconi; Pilar Camaño; Johannes G Dauwerse; Lauren Snider; Kirsten R Straasheijm; Gert Jan van Ommen; George W Padberg; Daniel G Miller; Stephen J Tapscott; Rabi Tawil; Rune R Frants; Silvère M van der Maarel
Journal:  Science       Date:  2010-08-19       Impact factor: 47.728

4.  The Dutch neuromuscular database CRAMP (Computer Registry of All Myopathies and Polyneuropathies): development and preliminary data.

Authors:  B G M van Engelen; H van Veenendaal; P A van Doorn; C G Faber; J H van der Hoeven; N G Janssen; N C Notermans; I N van Schaik; L H Visser; J J G M Verschuuren
Journal:  Neuromuscul Disord       Date:  2006-12-01       Impact factor: 4.296

5.  Facioscapulohumeral muscular dystrophy in the Dutch population.

Authors:  G W Padberg; R R Frants; O F Brouwer; C Wijmenga; E Bakker; L A Sandkuijl
Journal:  Muscle Nerve Suppl       Date:  1995

6.  Prevalence, incidence and duration.

Authors:  J Freeman; G B Hutchison
Journal:  Am J Epidemiol       Date:  1980-11       Impact factor: 4.897

  6 in total
  132 in total

Review 1.  Long-read sequencing for rare human genetic diseases.

Authors:  Satomi Mitsuhashi; Naomichi Matsumoto
Journal:  J Hum Genet       Date:  2019-09-27       Impact factor: 3.172

2.  Double SMCHD1 variants in FSHD2: the synergistic effect of two SMCHD1 variants on D4Z4 hypomethylation and disease penetrance in FSHD2.

Authors:  Marlinde L van den Boogaard; Richard J F L Lemmers; Pilar Camaño; Patrick J van der Vliet; Nicol Voermans; Baziel G M van Engelen; Adolfo Lopez de Munain; Stephen J Tapscott; Nienke van der Stoep; Rabi Tawil; Silvère M van der Maarel
Journal:  Eur J Hum Genet       Date:  2015-03-18       Impact factor: 4.246

Review 3.  Facioscapulohumeral muscular dystrophy (FSHD) molecular diagnosis: from traditional technology to the NGS era.

Authors:  Stefania Zampatti; Luca Colantoni; Claudia Strafella; Rosaria Maria Galota; Valerio Caputo; Giulia Campoli; Giulia Pagliaroli; Stefania Carboni; Julia Mela; Cristina Peconi; Stefano Gambardella; Raffaella Cascella; Emiliano Giardina
Journal:  Neurogenetics       Date:  2019-03-25       Impact factor: 2.660

4.  MRI-informed muscle biopsies correlate MRI with pathology and DUX4 target gene expression in FSHD.

Authors:  Leo H Wang; Seth D Friedman; Dennis Shaw; Lauren Snider; Chao-Jen Wong; Chris B Budech; Sandra L Poliachik; Nancy E Gove; Leann M Lewis; Amy E Campbell; Richard J F L Lemmers; Silvère M Maarel; Stephen J Tapscott; Rabi N Tawil
Journal:  Hum Mol Genet       Date:  2019-02-01       Impact factor: 6.150

5.  Dynamic transcriptomic analysis reveals suppression of PGC1α/ERRα drives perturbed myogenesis in facioscapulohumeral muscular dystrophy.

Authors:  Christopher R S Banerji; Maryna Panamarova; Johanna Pruller; Nicolas Figeac; Husam Hebaishi; Efthymios Fidanis; Alka Saxena; Julian Contet; Sabrina Sacconi; Simone Severini; Peter S Zammit
Journal:  Hum Mol Genet       Date:  2019-04-15       Impact factor: 6.150

6.  Cis D4Z4 repeat duplications associated with facioscapulohumeral muscular dystrophy type 2.

Authors:  Richard J L F Lemmers; Patrick J van der Vliet; Jeroen P Vreijling; Don Henderson; Nienke van der Stoep; Nicol Voermans; Baziel van Engelen; Frank Baas; Sabrina Sacconi; Rabi Tawil; Silvère M van der Maarel
Journal:  Hum Mol Genet       Date:  2018-10-15       Impact factor: 6.150

7.  Inter-individual differences in CpG methylation at D4Z4 correlate with clinical variability in FSHD1 and FSHD2.

Authors:  Richard J L F Lemmers; Jelle J Goeman; Patrick J van der Vliet; Merlijn P van Nieuwenhuizen; Judit Balog; Marianne Vos-Versteeg; Pilar Camano; Maria Antonia Ramos Arroyo; Ivonne Jerico; Mark T Rogers; Daniel G Miller; Meena Upadhyaya; Jan J G M Verschuuren; Adolfo Lopez de Munain Arregui; Baziel G M van Engelen; George W Padberg; Sabrina Sacconi; Rabi Tawil; Stephen J Tapscott; Bert Bakker; Silvère M van der Maarel
Journal:  Hum Mol Genet       Date:  2014-09-25       Impact factor: 6.150

Review 8.  Facioscapulohumeral Dystrophy.

Authors:  Leo H Wang; Rabi Tawil
Journal:  Curr Neurol Neurosci Rep       Date:  2016-07       Impact factor: 5.081

9.  Clinically Advanced p38 Inhibitors Suppress DUX4 Expression in Cellular and Animal Models of Facioscapulohumeral Muscular Dystrophy.

Authors:  Jonathan Oliva; Scott Galasinski; Amelia Richey; Amy E Campbell; Marvin J Meyers; Neal Modi; Jun Wen Zhong; Rabi Tawil; Stephen J Tapscott; Francis M Sverdrup
Journal:  J Pharmacol Exp Ther       Date:  2019-06-12       Impact factor: 4.030

10.  A multinational study on motor function in early-onset FSHD.

Authors:  Jean K Mah; Jia Feng; Marni B Jacobs; Tina Duong; Kate Carroll; Katy de Valle; Cara L Carty; Lauren P Morgenroth; Michela Guglieri; Monique M Ryan; Paula R Clemens; Mathula Thangarajh; Richard Webster; Edward Smith; Anne M Connolly; Craig M McDonald; Peter Karachunski; Mar Tulinius; Amy Harper; Avital Cnaan; Yi-Wen Chen
Journal:  Neurology       Date:  2018-03-14       Impact factor: 9.910

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