Literature DB >> 14648146

Oculopharyngeal muscular dystrophy with limb girdle weakness as major complaint.

Barbara M Van Der Sluijs1, Lies H Hoefsloot, George W Padberg, Silvère M Van Der Maarel, Baziel G M Van Engelen.   

Abstract

This first description of the oculopharyngeal muscular dystrophy (OPMD) phenotype in Dutch patients shows that limb girdle weakness can occur early in the course of disease and can give the first and major complaint in OPMD patients. The aim of this study was to examine clinically, histologically and genetically all Dutch OPMD patients known at the Neuromuscular Centre Nijmegen, to measure the limb girdle weakness (MRC scale) and to quantify the consequences of the limb girdle weakness on daily activities (Rankin scale). Remarkable in this population was the early onset and the severity of the limb girdle weakness. We found a higher percentage of patients with limb girdle weakness than reported before in non-Dutch OPMD populations. This limb girdle weakness caused limitations in daily activities more than the other symptoms of OPMD. It was difficult to compare the severity of the limb girdle weakness of Dutch patients with other patients because of the lack of data related to quantification of limb girdle weakness in non-Dutch OPMD patients. Because of the influence of the limb girdle weakness on the daily activities of the patients, we recommend that more attention is paid to the proximal limb muscles in OPMD patients early in the course of the disease.

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Year:  2003        PMID: 14648146     DOI: 10.1007/s00415-003-0201-6

Source DB:  PubMed          Journal:  J Neurol        ISSN: 0340-5354            Impact factor:   4.849


  13 in total

1.  Unique PABPN1 gene mutation in a large Bulgarian family with OPMD.

Authors:  V Mihaylova; T Müller; I Petrova; I Tournev; S Cherninkova; M C Walter; M Deschauer
Journal:  J Neurol       Date:  2008-02-19       Impact factor: 4.849

2.  Hip flexion weakness is associated with impaired mobility in oculopharyngeal muscular dystrophy: a retrospective study with implications for trial design.

Authors:  Sarah Youssof; Ronald Schrader; David Bear; Leslie Morrison
Journal:  Neuromuscul Disord       Date:  2014-11-20       Impact factor: 4.296

3.  Novel mouse models of oculopharyngeal muscular dystrophy (OPMD) reveal early onset mitochondrial defects and suggest loss of PABPN1 may contribute to pathology.

Authors:  Katherine E Vest; Brittany L Phillips; Ayan Banerjee; Luciano H Apponi; Eric B Dammer; Weiting Xu; Dinghai Zheng; Julia Yu; Bin Tian; Grace K Pavlath; Anita H Corbett
Journal:  Hum Mol Genet       Date:  2017-09-01       Impact factor: 6.150

4.  A Preliminary Videofluoroscopic Investigation of Swallowing Physiology and Function in Individuals with Oculopharyngeal Muscular Dystrophy (OPMD).

Authors:  Ashley A Waito; Catriona M Steele; Melanie Peladeau-Pigeon; Angela Genge; Zohar Argov
Journal:  Dysphagia       Date:  2018-05-03       Impact factor: 3.438

5.  The NIH Office of Rare Diseases Research patient registry Standard: a report from the University of New Mexico's Oculopharyngeal Muscular Dystrophy Patient Registry.

Authors:  Shamsi Daneshvari; Sarah Youssof; Philip J Kroth
Journal:  AMIA Annu Symp Proc       Date:  2013-11-16

Review 6.  PABPN1: molecular function and muscle disease.

Authors:  Ayan Banerjee; Luciano H Apponi; Grace K Pavlath; Anita H Corbett
Journal:  FEBS J       Date:  2013-05-24       Impact factor: 5.542

7.  Loss of nuclear poly(A)-binding protein 1 causes defects in myogenesis and mRNA biogenesis.

Authors:  Luciano H Apponi; Sara W Leung; Kathryn R Williams; Sandro R Valentini; Anita H Corbett; Grace K Pavlath
Journal:  Hum Mol Genet       Date:  2009-12-24       Impact factor: 6.150

8.  A decline in PABPN1 induces progressive muscle weakness in oculopharyngeal muscle dystrophy and in muscle aging.

Authors:  Seyed Yahya Anvar; Yotam Raz; Nisha Verway; Barbara van der Sluijs; Andrea Venema; Jelle J Goeman; John Vissing; Silvère M van der Maarel; Peter A C 't Hoen; Baziel G M van Engelen; Vered Raz
Journal:  Aging (Albany NY)       Date:  2013-06       Impact factor: 5.682

9.  Nuclear entrapment and extracellular depletion of PCOLCE is associated with muscle degeneration in oculopharyngeal muscular dystrophy.

Authors:  Vered Raz; Ellen Sterrenburg; Samantha Routledge; Andrea Venema; Barbara M van der Sluijs; Capucine Trollet; George Dickson; Baziel G M van Engelen; Silvère M van der Maarel; Michael N Antoniou
Journal:  BMC Neurol       Date:  2013-07-01       Impact factor: 2.474

10.  Control of mRNA stability contributes to low levels of nuclear poly(A) binding protein 1 (PABPN1) in skeletal muscle.

Authors:  Luciano H Apponi; Anita H Corbett; Grace K Pavlath
Journal:  Skelet Muscle       Date:  2013-10-01       Impact factor: 4.912

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