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First long-term experience with the orphan drug rufinamide in children with myoclonic-astatic epilepsy (Doose syndrome).
MedLine Citation:
PMID:  22266062     Owner:  NLM     Status:  Publisher    
INTRODUCTION: We evaluated the long-term efficacy and tolerability of the orphan drug rufinamide (RUF) in children with pharmacoresistant myoclonic-astatic epilepsy (MAE, Doose syndrome). METHODS: This was a retrospective European multicenter study on eight patients who had started an intention-to-treat trial of RUF between July 2007 and June 2010. Clinical information was collected via questionnaire. Responder rate was defined as reduction of seizure frequency ≥50% in comparison to four weeks before starting RUF. Maximum follow-up was eighteen months. RESULTS: Responder rates were 7/8 patients after 3 months, 6/8 patients after 6 months and 5/8 patients after 12 months. RUF seemed particularly effective in the prevention of myoclonic-astatic seizures (comparable with drop attacks in Lennox-Gastaut-Syndrome, for which RUF is particularly effective). Some loss of efficacy was noticed in the long-term observation. Side-effects occurred in two patients. Seizure aggravation was not observed. CONCLUSION: RUF seems to be a promising therapeutic option in children with MAE. Further studies are warranted to confirm these first observations.
C von Stülpnagel; G Coppola; P Striano; A Müller; M Staudt; G Kluger
Publication Detail:
Type:  JOURNAL ARTICLE     Date:  2012-1-20
Journal Detail:
Title:  European journal of paediatric neurology : EJPN : official journal of the European Paediatric Neurology Society     Volume:  -     ISSN:  1532-2130     ISO Abbreviation:  -     Publication Date:  2012 Jan 
Date Detail:
Created Date:  2012-1-23     Completed Date:  -     Revised Date:  -    
Medline Journal Info:
Nlm Unique ID:  9715169     Medline TA:  Eur J Paediatr Neurol     Country:  -    
Other Details:
Languages:  ENG     Pagination:  -     Citation Subset:  -    
Copyright Information:
Copyright © 2011 European Paediatric Neurology Society. Published by Elsevier Ltd. All rights reserved.
Clinic for Neuropediatrics and Neurological Rehabilitation, Epilepsy-Center for Children and Adolescents, Vogtareuth, Germany.
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