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Successful use of albuterol in a patient with central core disease and mitochondrial dysfunction.
MedLine Citation:
PMID:  20443062     Owner:  NLM     Status:  Publisher    
Abstract/OtherAbstract:
Albuterol, a selective beta-adrenergic agonist, has been used experimentally in combination with exercise therapy in a few inherited neuromuscular disorders to increase muscle strength and muscle volume . We report on a 9-year-old boy with central core disease and mitochondrial dysfunction due to compound heterozygous RYR1 mutations receiving albuterol treatment for 1 year. Throughout the period of albuterol administration, the patient underwent an aerobic exercise regime of training sessions three times a week that lasted 20 min each. No side effects of albuterol use were seen. Significant clinical progress, including self care, sitting up, raising arms above the shoulders, independent feeding, and better speech and writing were observed compared with minimal development of these abilities in the previous years on physiotherapy. Improved forced expiratory volume in 1 s (FEV(1)) score was detected and increased muscle strength was noted: progress was measured using various functional tests and assessment scales. The only complication observed was a mild progression of the joint contractures, possibly due to an unbalance between the flexor and extensor musculature. In general, in this pilot study in a complex case of metabolic myopathy our patient has shown promising results following albuterol treatment and aerobic exercise therapy.
Authors:
L T W Schreuder; M W G Nijhuis-van der Sanden; A de Hair; G Peters; S Wortmann; L A Bok; E Morava
Publication Detail:
Type:  JOURNAL ARTICLE     Date:  2010-5-5
Journal Detail:
Title:  Journal of inherited metabolic disease     Volume:  -     ISSN:  1573-2665     ISO Abbreviation:  -     Publication Date:  2010 May 
Date Detail:
Created Date:  2010-5-5     Completed Date:  -     Revised Date:  -    
Medline Journal Info:
Nlm Unique ID:  7910918     Medline TA:  J Inherit Metab Dis     Country:  -    
Other Details:
Languages:  ENG     Pagination:  -     Citation Subset:  -    
Affiliation:
Departments of Pediatrics and Neurology, Nijmegen Centre for Mitochondrial Disorders, Radboud University Nijmegen Medical Centre, P.O Box 9101, 6500, HB, Nijmegen, The Netherlands.
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