TY - JOUR A1 - Walter, Maggie C. A1 - Reilich, Peter A1 - Thiele, Simone A1 - Schessl, Joachim A1 - Schreiber, Herbert A1 - Reiners, Karlheinz A1 - Kress, Wolfram A1 - Müller-Reible, Clemens A1 - Vorgerd, Matthias A1 - Urban, Peter A1 - Schrank, Bertold A1 - Deschauer, Marcus A1 - Schlotter-Weigel, Beate A1 - Kohnen, Ralf A1 - Lochmüller, Hans T1 - Treatment of dysferlinopathy with deflazacort: a double-blind, placebo-controlled clinical trial JF - Orphanet Journal of Rare Diseases N2 - Background: Dysferlinopathies are autosomal recessive disorders caused by mutations in the dysferlin (DYSF) gene encoding the dysferlin protein. DYSF mutations lead to a wide range of muscular phenotypes, with the most prominent being Miyoshi myopathy (MM) and limb girdle muscular dystrophy type 2B (LGMD2B). Methods: We assessed the one-year-natural course of dysferlinopathy, and the safety and efficacy of deflazacort treatment in a double-blind, placebo-controlled cross-over trial. After one year of natural course without intervention, 25 patients with genetically defined dysferlinopathy were randomized to receive deflazacort and placebo for six months each (1 mg/kg/day in month one, 1 mg/kg every 2nd day during months two to six) in one of two treatment sequences. Results: During one year of natural course, muscle strength declined about 2% as measured by CIDD (Clinical Investigation of Duchenne Dystrophy) score, and 76 Newton as measured by hand-held dynamometry. Deflazacort did not improve muscle strength. In contrast, there is a trend of worsening muscle strength under deflazacort treatment, which recovers after discontinuation of the study drug. During deflazacort treatment, patients showed a broad spectrum of steroid side effects. Conclusion: Deflazacort is not an effective therapy for dysferlinopathies, and off-label use is not warranted. This is an important finding, since steroid treatment should not be administered in patients with dysferlinopathy, who may be often misdiagnosed as polymyositis. KW - Deflazacort KW - muscle strength KW - gridle muscular-dystrophy KW - Duchenne dystrphy KW - Miyoshi myopathy KW - mutation KW - prednisone KW - gene KW - 2B KW - children KW - design KW - steroids KW - therapy KW - dysferlinopathy KW - Limb girdle muscular dystrophy (LGMD) Y1 - 2013 U6 - http://nbn-resolving.de/urn/resolver.pl?urn:nbn:de:bvb:20-opus-125663 SN - 1750-1172 VL - 8 IS - 26 ER - TY - JOUR A1 - Krüger, H.-P. A1 - Bonerad, E. M. A1 - Dennler, H. J. A1 - Dunkl, E. A1 - Friese, H. J. A1 - Hain, C. A1 - Kazenwadel, J. A1 - Kohnen, Ralf A1 - Maier, W. A1 - Menzel, M. T1 - Speech chronemics in drug evaluation N2 - No abstract available Y1 - 1992 U6 - http://nbn-resolving.de/urn/resolver.pl?urn:nbn:de:bvb:20-opus-41213 ER - TY - JOUR A1 - Kohnen, Ralf A1 - Krüger, Hans-Peter T1 - Drug Effects on Human Social Behavior: Chances in Talking Activities Induced by CGP 361/A, a Beta-blocking Agent N2 - No abstract available Y1 - 1986 U6 - http://nbn-resolving.de/urn/resolver.pl?urn:nbn:de:bvb:20-opus-41228 ER - TY - JOUR A1 - Kohnen, Ralf A1 - Krüger, Hans-Peter T1 - Tranquillizer effects in an experimental analog of verbal examinations N2 - In an experimental analog of verbal examinations, the call-up situation, the effects of two dosages of a tranquillizing agent (lopirazepam) are compared to placebo treatment. 72 male and female, healthy, young volunteers have been randomly assigned to 12 groups of 6 subjects each. Pulse frequency and performance were registered. The results indicated differential drug effects which were interpreted according to the hypotheses of "differential effects of social stressors". If a situation was highly challenging for a subject, the application of a tranquillizer in an adequately high dosage enabled him to perform well in spite of or because of strong increases in pulse frequency. KW - Tranquillizer KW - Social stress KW - Examination KW - Healthy subjects KW - Differential effects of stressors KW - Telemetry KW - Activation Y1 - 1982 U6 - http://nbn-resolving.de/urn/resolver.pl?urn:nbn:de:bvb:20-opus-41617 ER - TY - JOUR A1 - Krüger, Hans-Peter A1 - Kohnen, Ralf T1 - Tranquillizer effects in an experimental analog of group psychotherapy N2 - In an experimental analog of verbal examinations, the call-up situation, the effects of two dosages of a tranquillizing agent (lopirazepam) are compared to placebo treatment. 72 male and female, healthy, young volunteers have been randomly assigned to 12 groups of 6 subjects each. Pulse frequency and performance were registered. The results indicated differential drug effects which were interpreted according to the hypotheses of "differential effects of social stressors". If a situation was highly challenging for a subject, the application of a tranquillizer in an adequately high dosage enabled him to perform well in spite of or because of strong increases in pulse frequency. KW - Tranquillizer KW - Social stress situations KW - Group dynamics KW - Healthy subjects KW - Differential effects of stressors KW - Anxiolysis KW - Telemetry Activation Y1 - 1982 U6 - http://nbn-resolving.de/urn/resolver.pl?urn:nbn:de:bvb:20-opus-41316 ER -