Literature DB >> 16887341

Current treatment of adult Duchenne muscular dystrophy.

Kathryn R Wagner1, Noah Lechtzin, Daniel P Judge.   

Abstract

Patients with Duchenne muscular dystrophy (DMD) are living longer into adulthood due to a variety of improvements in health care practices. This growing patient population presents new therapeutic challenges. In this article, we review the literature on current treatment of adult DMD as well as our own experience as a multidisciplinary team actively caring for 23 men ages 19-38 years of age. Approximately one quarter of our adult DMD patients have remained on moderate dose corticosteroids. Daily stretching exercises are recommended, particularly of the distal upper extremities. Cardiomyopathy is anticipated, detected, and treated early with afterload reduction. Oxygen saturation monitoring, noninvasive positive pressure ventilation and cough assist devices are routinely used. Other medical issues such as osteoporosis, gastrointestinal and urinary symptoms are addressed. Current and future therapies directed at prolonging the lifespan of those with DMD will result in further increases in this adult population with special needs and concerns. These needs are best addressed in a multidisciplinary clinic.

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Year:  2006        PMID: 16887341     DOI: 10.1016/j.bbadis.2006.06.009

Source DB:  PubMed          Journal:  Biochim Biophys Acta        ISSN: 0006-3002


  46 in total

1.  Focal but reversible diastolic sheet dysfunction reflects regional calcium mishandling in dystrophic mdx mouse hearts.

Authors:  Ya-Jian Cheng; Di Lang; Shelton D Caruthers; Igor R Efimov; Junjie Chen; Samuel A Wickline
Journal:  Am J Physiol Heart Circ Physiol       Date:  2012-07-09       Impact factor: 4.733

2.  Delta-sarcoglycan gene therapy halts progression of cardiac dysfunction, improves respiratory failure, and prolongs life in myopathic hamsters.

Authors:  Masahiko Hoshijima; Takeharu Hayashi; Young E Jeon; Zhenxing Fu; Yusu Gu; Nancy D Dalton; Mark H Ellisman; Xiao Xiao; Frank L Powell; John Ross
Journal:  Circ Heart Fail       Date:  2010-10-29       Impact factor: 8.790

Review 3.  Approaching a new age in Duchenne muscular dystrophy treatment.

Authors:  Kathryn R Wagner
Journal:  Neurotherapeutics       Date:  2008-10       Impact factor: 7.620

4.  Effective rescue of dystrophin improves cardiac function in dystrophin-deficient mice by a modified morpholino oligomer.

Authors:  Bo Wu; Hong M Moulton; Patrick L Iversen; Jiangang Jiang; Juan Li; Jianbin Li; Christopher F Spurney; Arpana Sali; Alfredo D Guerron; Kanneboyina Nagaraju; Timothy Doran; Peijuan Lu; Xiao Xiao; Qi Long Lu
Journal:  Proc Natl Acad Sci U S A       Date:  2008-09-19       Impact factor: 11.205

5.  Sildenafil does not improve cardiomyopathy in Duchenne/Becker muscular dystrophy.

Authors:  Doris G Leung; Daniel A Herzka; W Reid Thompson; Bing He; Genila Bibat; Gihan Tennekoon; Stuart D Russell; Karl H Schuleri; Albert C Lardo; David A Kass; Richard E Thompson; Daniel P Judge; Kathryn R Wagner
Journal:  Ann Neurol       Date:  2014-07-10       Impact factor: 10.422

Review 6.  Diagnosis and management of Duchenne muscular dystrophy, part 3: primary care, emergency management, psychosocial care, and transitions of care across the lifespan.

Authors:  David J Birnkrant; Katharine Bushby; Carla M Bann; Susan D Apkon; Angela Blackwell; Mary K Colvin; Linda Cripe; Adrienne R Herron; Annie Kennedy; Kathi Kinnett; James Naprawa; Garey Noritz; James Poysky; Natalie Street; Christina J Trout; David R Weber; Leanne M Ward
Journal:  Lancet Neurol       Date:  2018-02-02       Impact factor: 44.182

Review 7.  Gene therapy in large animal models of muscular dystrophy.

Authors:  Zejing Wang; Jeffrey S Chamberlain; Stephen J Tapscott; Rainer Storb
Journal:  ILAR J       Date:  2009

8.  Leaky RyR2 trigger ventricular arrhythmias in Duchenne muscular dystrophy.

Authors:  Jérémy Fauconnier; Jérôme Thireau; Steven Reiken; Cécile Cassan; Sylvain Richard; Stefan Matecki; Andrew R Marks; Alain Lacampagne
Journal:  Proc Natl Acad Sci U S A       Date:  2010-01-04       Impact factor: 11.205

9.  The chondrogenic response to exercise in the proximal femur of normal and mdx mice.

Authors:  David J Nye; Jeffrey M Costas; Jessica B Henley; Jin-Kwang Kim; Jeffrey H Plochocki
Journal:  BMC Musculoskelet Disord       Date:  2010-09-03       Impact factor: 2.362

10.  Octa-guanidine morpholino restores dystrophin expression in cardiac and skeletal muscles and ameliorates pathology in dystrophic mdx mice.

Authors:  Bo Wu; Yongfu Li; Paul A Morcos; Timothy J Doran; Peijuan Lu; Qi Long Lu
Journal:  Mol Ther       Date:  2009-03-10       Impact factor: 11.454

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