@article{WalterReilichThieleetal.2013, author = {Walter, Maggie C. and Reilich, Peter and Thiele, Simone and Schessl, Joachim and Schreiber, Herbert and Reiners, Karlheinz and Kress, Wolfram and M{\"u}ller-Reible, Clemens and Vorgerd, Matthias and Urban, Peter and Schrank, Bertold and Deschauer, Marcus and Schlotter-Weigel, Beate and Kohnen, Ralf and Lochm{\"u}ller, Hans}, title = {Treatment of dysferlinopathy with deflazacort: a double-blind, placebo-controlled clinical trial}, series = {Orphanet Journal of Rare Diseases}, volume = {8}, journal = {Orphanet Journal of Rare Diseases}, number = {26}, issn = {1750-1172}, doi = {10.1186/1750-1172-8-26}, url = {http://nbn-resolving.de/urn:nbn:de:bvb:20-opus-125663}, year = {2013}, abstract = {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.}, language = {en} } @article{KruegerKohnen1982, author = {Kr{\"u}ger, Hans-Peter and Kohnen, Ralf}, title = {Tranquillizer effects in an experimental analog of group psychotherapy}, url = {http://nbn-resolving.de/urn:nbn:de:bvb:20-opus-41316}, year = {1982}, abstract = {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.}, language = {en} } @article{KruegerBoneradDennleretal.1992, author = {Kr{\"u}ger, H.-P. and Bonerad, E. M. and Dennler, H. J. and Dunkl, E. and Friese, H. J. and Hain, C. and Kazenwadel, J. and Kohnen, Ralf and Maier, W. and Menzel, M.}, title = {Speech chronemics in drug evaluation}, url = {http://nbn-resolving.de/urn:nbn:de:bvb:20-opus-41213}, year = {1992}, abstract = {No abstract available}, language = {en} } @article{KohnenKrueger1982, author = {Kohnen, Ralf and Kr{\"u}ger, Hans-Peter}, title = {Tranquillizer effects in an experimental analog of verbal examinations}, url = {http://nbn-resolving.de/urn:nbn:de:bvb:20-opus-41617}, year = {1982}, abstract = {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.}, language = {en} } @article{KohnenKrueger1986, author = {Kohnen, Ralf and Kr{\"u}ger, Hans-Peter}, title = {Drug Effects on Human Social Behavior: Chances in Talking Activities Induced by CGP 361/A, a Beta-blocking Agent}, url = {http://nbn-resolving.de/urn:nbn:de:bvb:20-opus-41228}, year = {1986}, abstract = {No abstract available}, language = {en} }