Bei der Duchenne Muskeldystrophie (DMD) handelt es sich um eine erblich bedingte, progredient verlaufende Muskelschwäche mit frühzeitigem tödlichem Ausgang. Durch eine Mutation des Dystrophingens kommt es aufgrund fehlender mechanischer Stabilität der Muskelzellen zur Muskelschwäche und dem Ersatz von Muskelgewebe durch Binde- und Fettgewebe. Ein weiteres Kennzeichen der Erkrankung ist ein pathologisch erhöhter Kalziumgehalt der Muskelzellen, der zu gestörten Signalwegen und dem Zelltod führt. Die DMD kommt auch bei Mäusen vor, wobei das Mdx-Mausmodell das bekannteste und am besten erforschte Modell der Erkrankung ist. Die Mäuse besitzen im Gegensatz zum Menschen allerdings ein De- und Regenerationspotenzial der Muskulatur, was die Übertragbarkeit der Ergebnisse auf die menschliche DMD erschwert, da sich die Pathophysiologie der Mäuse abgemildert darstellt. Daher ist es notwendig, das Modell hinsichtlich der Degeneration der Muskulatur zu verbessern, was durch eine unilaterale Injektion von Botox in den rechten M.masseter, die zu dessen Lähmung führt, gewährleistet werden sollte. Das vom Bakterium Clostridium botulinum produzierte Endotoxin Botulinumtoxin A (Botox) wird in der Zahnmedizin unter anderem eingesetzt, um die Masseterhypertrophie zu behandeln. Botox hemmt die Freisetzung von Acetylcholin an der neuromuskulären Endplatte, was zur Hemmung der Muskelkontraktion führt, welche beendet wird, wenn durch das Sprossen neuer Axone eine Reinnervation der Muskulatur stattfindet. Unter dem Einfluss von Botox zu unterschiedlichen Untersuchungszeitpunkten sollte in dieser Arbeit gezeigt werden, dass sich die Expression der kalziumregulierenden Gene der Mdx-Mäuse dahingehend verändern, dass ein verschlechterter Phänotyp bei den dystrophen Mäusen resultiert. Das Mdx-Mausmodell sollte der menschlichen DMD angepasst werden, indem das regenerative Potenzial der dystrophen Mäuse durch die Botox-Injektion und dem damit verbundenen unilateralen Funktionsverlust der Muskulatur unterbrochen wird. Weiterhin stellte sich die Frage, ob durch die Botox-Injektion auch bei den gesunden Mäusen ein Duchenne-ähnlicher Phänotyp auf der behandelten Seite erzeugt werden kann. Dazu wurden in dieser Arbeit die Kaumuskeln M.masseter, M.temporalis und die Zunge von 100 Tage alten Kontroll- und Mdx-Mäusen hinsichtlich ihres Kalziumstoffwechsels 3 bzw. 6 Wochen nach der Botox-Injektion untersucht. Die Genexpression der untersuchten Gene wurde durch quantitative RT-PCR erhoben. Bei der Kontrollgruppe waren 3 Wochen nach der Botox-Injektion im rechten M.masseter alle untersuchten Gene mit Ausnahme von Atp2a1 und Pvalb erhöht. Die erhöhten Expressionen im Vergleich zur linken Seite lagen zwischen dem 1,7-und 15,3fachen. Sechs Wochen nach der Botox-Injektion waren alle Gene im rechten M.masseter mit Ausnahme von Atp2a2 und Sln zwischen dem 1,9- und 4,6fachen erhöht. Bei Sln kam es zu einer Reduktion um 88% verglichen mit der linken Seite. Im M.temporalis der Kontrolltiere konnten 3 Wochen nach der Injektion die Gene Slc8a1 und Sln um das 1,8- bzw. 13,2fache erhöht im Vergleich zur linken Seite nachgewiesen werden. Atp2a1 wurde im rechten M.temporalis um 47% weniger exprimiert als auf der linken Seite. Sechs Wochen nach der Injektion konnten im M.temporalis keine Veränderungen mehr festgestellt werden. Bei den Mdx-Mäusen wurde 3 Wochen nach der Botox-Injektion im rechten M.masseter bei den Genen Atp2a2, Pln und Sln erhöhte Genexpressionen um das Doppelte, 2,2- und 2,8fache im Vergleich zur linken Seite nachgewiesen. Im M.temporalis kam es 3 Wochen nach der Injektion auf der rechten Seite zu einer Erhöhung von Sln um das 8,5fache im Vergleich zur linken Seite. Sechs Wochen nach der Injektion gab es in beiden rechten Muskeln keine Veränderungen mehr. Bei den gesunden Mäusen konnte durch die Botox-Injektion zu keinem der beiden Untersuchungszeitpunkte und in keiner der Muskelproben ein Duchenne-ähnlicher Phänotyp erzeugt werden, auch wenn einzelne Gene eine Verschlechterung des Phänotyps anzeigen, gleichzeitig andere Genveränderungen jedoch auf einen positiven Einfluss auf den Phänotyp schließen lassen. Die Funktion des M.temporalis gleicht außerdem den Funktionsverlust des M.masseter ausreichend aus. Bei den Mdx-Mäusen konnte im rechten M.masseter ebenfalls keine eindeutige Verschlechterung des Phänotyps 3 Wochen nach der Botox-Injektion nachgewiesen werden. Die veränderten Genexpressionen lassen sowohl positiven als auch negativen Einfluss auf den Phänotyp zu, sodass es in der Gesamtbetrachtung nicht zu einer Verschlechterung des Phänotyps kommen muss. Zum späteren Untersuchungszeitpunkt konnten bei den Mdx-Mäusen keinerlei Veränderungen hinsichtlich der Genexpression in den beiden Muskelproben nachgewiesen werden. Es ist davon auszugehen, dass die Botox-Wirkung nachgelassen bzw. aufgehört hat und es bereits zu einer Reinnervation im M.masseter kam, die auch durch das Regenerationspotenzial der Mdx-Mäuse erklärt werden kann. Auch in anderen Untersuchungen konnte der vielversprechende Ansatz einer durch Botox induzierten Muskellähmung nicht zu einer Verschlechterung des Phänotyps der Kaumuskeln bei gesunden und Mdx-Mäusen führen.:Abkürzungsverzeichnis Abbildungsverzeichnis Tabellenverzeichnis 1. Einleitung 1.1. Muskeldystrophie Duchenne 1.2. Das Dystrophinprotein 1.3. Der Kalziumstoffwechsel 1.4. Tiermodelle 1.4.1. Maus 1.4.2. Hund 1.4.3. Schwein 1.4.4. Ratte 1.5. Botulinumtoxin in der Zahnmedizin 1.6. Ziele der Arbeit 2. Material und Methoden 2.1. Material 2.1.1. Geräte 2.1.2. Chemikalien 2.1.3. Kits 2.1.4. Sonden/Primer 2.1.5. Software 2.2. Methoden 2.2.1. Versuchstiere und Probengewinnung 2.2.2. Gelelektrophorese 2.2.3. RNA-Gewinnung 2.2.4. cDNA-Synthese 2.2.5. Quantitative real time PCR (qRT-PCR) 2.2.6. Quantifizierungsmethoden 2.2.6.1. ΔΔCT-Methode 2.2.7. Statistische Auswertung 3. Ergebnisse 3.1. Gelelektrophorese 3.2. mRNA-Gehalt 3 Wochen nach Botox-Injektion 3.2.1. Expression von Atp2a1 3.2.2. Expression von Atp2a2 3.2.3. Expression von Atp2b4 3.2.4. Expression von Cacna1s 3.2.5. Expression von Pvalb 3.2.6. Expression von Pln 3.2.7. Expression von Ryr1 3.2.8. Expression von Slc8a1 3.2.9. Expression von Sln 3.3. mRNA-Gehalt 6 Wochen nach Botox-Injektion 3.3.1. Expression von Atp2a1 3.3.2. Expression von Atp2a2 3.3.3. Expression von Atp2b4 3.3.4. Expression von Cacna1s 3.3.5. Expression von Pvalb 3.3.6. Expression von Pln 3.3.7. Expression von Ryr1 3.3.8. Expression von Slc8a1 3.3.9. Expression von Sln 3.4. Vergleich der mRNA-Gehalte 3 Wochen und 6 Wochen nach der Botox-Injektion 3.4.1. Atp2a1 3.4.2. Atp2a2 3.4.3. Atp2b4 3.4.4. Cacna1s 3.4.5. Pvalb 3.4.6. Pln 3.4.7. Ryr1 3.4.8. Slc8a1 3.4.9. Sln 4. Diskussion 4.1. Erzeugung eines Duchenne-ähnlichen Phänotyps bei gesunden Mäusen durch Botox-Injektion 4.2. Verschlechterung des Phänotyps bei Mdx-Mäusen 4.3. Unterschiede zwischen den Untersuchungszeitpunkten 4.4. Schlussfolgerungen und kritische Auseinandersetzung mit dem Versuchsaufbau 5. Zusammenfassung 6. Summary 7. Literatur 8. Danksagung 9. Anlage 1: Erklärung zur Eröffnung des Promotionsverfahrens 10. Anlage 2: Erklärung zur Einhaltung aktueller gesetzlicher Vorgaben

No description available.

Drooling management in the neurologically impaired pediatric population is a challenge. Surgery is considered an invasive procedure, while behaviour modification techniques, correction of situational factors and oral-motor therapy do not always produce sustained improvement. In recent years Botox® has been investigated. This study comprised analyses of clinical data obtained from a Drooling Treatment Project (DTP) conducted at a school for special needs children in South Africa. The aims of the DTP were to establish the response of drooling in a number of different contexts, following bilateral submandibular salivary gland injections of Botox®. Two groups of children were involved, 7 children with cerebral palsy and 2 children with operculum syndrome. Drooling was assessed in 5 different situations and at different time points pre- and post Botox® injection up to 6 months. Parents’/primary caregivers’ perceptions of drooling and treatment with Botox® were also measured using an interview form and a quality of life questionnaire. Results showed that drooling was reduced in all situations, with significant reductions in the general and communicating situations. These results indicate that the context in which drooling occurs is an important factor and suggest the value of considering the situational context when making drooling judgements. Further, there was a difference in the pattern of response between the 2 groups. This finding has implications, not only for future research, but also for models of explanation of the effects of Botox®. Most parents/primary caregivers felt their children’s lives and their own had improved following the Botox® injection and would repeat the treatment. Clinical and research implications are discussed, with reference to the South African context.

INTRODUÇÃO: O objetivo do presente estudo é apresentar dados morfométricos da fenda palpebral e da função do músculo levantador da pálpebra superior, ao longo de 6 meses de seguimento, após a aplicação da toxina botulínica tipo A, em pacientes com retração palpebral nas fases aguda e crônica da orbitopatia distireoidiana. MÉTODOS: Estudo prospectivo, no qual foram incluídos 24 indivíduos, divididos em dois grupos, agudos (n=12) e crônicos (n=12). Foram submetidos à aplicação de toxina botulínica na dose de 5 UI/0,1 ml em uma das pálpebras superiores e seguidos por 6 meses. A avaliação palpebral foi registrada por meio de captação de imagens com uma câmera filmadora de vídeo conectada a um microcomputador. Os valores médios obtidos nos diferentes momentos analisados foram comparados entre os dois grupos. RESULTADOS: A maioria dos pacientes apresentou melhora da retração da pálpebra superior, com redução média de 3,05 mm no grupo agudo e 3,81 mm no grupo crônico. Esta redução foi significativa até 1 mês no grupo agudo e até 3 meses no grupo crônico. Houve diminuição da função do músculo levantador e da diferença entre as áreas lateral e medial da fenda palpebral nos olhos tratados em ambos os grupos. No olho contralateral, houve aumento significativo da fenda palpebral até 2 semanas após a aplicação. Não houve diferença na freqüência dos efeitos colaterais entre os 2 grupos. CONCLUSÕES: A injeção de 5 unidades foi eficaz na redução da retração e da função do músculo levantador e na melhora do contorno palpebral superior, nos pacientes na fase aguda e crônica da orbitopatia distireoidiana, embora esta melhora tenha sido menos duradoura no grupo agudo / INTRODUCTION: The objective of this study is to compare morphometric data of the eyelid fissure and the levator muscle function after transcutaneous injection of botulinum toxin type A with six-month follow-up in patients with upper eyelid retraction from acute and chronic dysthyroid orbitopathy. METHODS: This was a prospective study that included 24 individuals with dysthyroid orbitopathy, divided into two groups, acute (n=12) and chronic (n=12). They were given a 5 UI/0,1 ml dose of botulinum toxin in the upper eyelid and examined periodically for 6 months. Clinical eyelid examination was performed and recorded using a video camera connected to a microcomputer. Mean values taken at different follow-up points were compared for the two groups. RESULTS: Most patients experienced marked improvement in upper eyelid retraction, with a mean reduction of 3.05 mm in acute group and 3.81 mm in chronic group. This reduction was significantly smaller during 1 month in acute group and during 3 months in chronic group. Reduction in levator function and in the difference between lateral and medial lid fissure measurements was observed in both groups. Measurements of the contralateral eye increased up to 2 weeks after the application. No difference was observed between the study groups with regard to the observed side effects. CONCLUSIONS: A single 5-unit botulinum toxin injection improved upper eyelid retraction, reduced levator function, and produced an adequate lid contour in patients with acute and chronic dysthyroid orbitopathy. The effect lasts longer in patients with chronic disease than in patients with acute disease.

INTRODUÇÃO: A paralisia facial é constrangedora tanto do ponto de vista funcional quanto estético. Esta pesquisa teve por objetivo verificar o efeito da terapia miofuncional em pacientes com paralisia facial de longa duração associada à aplicação de toxina botulínica. MÉTODOS: Foram tratados vinte e cinco pacientes, divididos em dois grupos. Os pacientes do grupo A receberam quatro sessões de terapia miofuncional antes da aplicação de toxina botulínica e os pacientes do grupo B simultaneamente à aplicação. A terapia foi composta por manobras isométricas e isotônicas passivas, intra e extraorais, além de exercícios de resistência. RESULTADOS: Após a terapia miofuncional os pacientes apresentaram aumento significativo da mobilidade do lado paralisado da face, do índice de satisfação do paciente com a face, do Índice Funcional da Face (IFF) e do Índice de Bem-Estar Social (IBES). O grupo de pacientes que realizaram a terapia miofuncional previamente, apresentou freqüência significativamente maior de dificuldade para falar, enquanto o grupo que realizou a terapia miofuncional a partir da data da aplicação de toxina botulínica, apresentou freqüência significativamente maior de dificuldade para mastigar. A terapia miofuncional promove simetria facial; satisfação dos pacientes com a face, funcionalidade oromiofacial, qualidade de vida e deve ser realizada antes e após aplicação de toxina botulínica para reduzir os possíveis efeitos adversos. / INTRODUCTION: The facial paralysis is constraining so much of the functional point of view as aesthetic. This research had for goal verified the myofunctional therapy effect in patients with long-standing facial paralysis associate to the botulinum toxin application. METHODS: Twenty-five patients were treated, divided into two groups. The patients from the group A received four sessions of myofunctional therapy before the toxin botulinum application and the patients from the group B received it simultaneously to the application. The therapy was composed by isometric and passive isotonic maneuvers, inside and outside oral, and resistance exercises. RESULTS: After the myofuncional therapy the patients presented significant increase of the mobility of the paralyzed side, of the patient satisfaction index with the face, Functional Index of the Face (IFF) and of the Index of Social Welfare (IBES). The group of patient that accomplished the myofuncional therapy previously presented significantly larger frequency of talking difficulty, while the group that accomplished the miofuncional therapy from the toxin botulínica application date presented significantly larger frequency of chewing difficulty. The myofuncional therapy promotes facial symmetry; patients satisfaction with the face, myofuncional functionality, life quality, and should be accomplished before and after toxin botulínica application to reduce the possible adverse effects.

Made available in DSpace on 2017-07-21T14:13:12Z (GMT). No. of bitstreams: 1 Daniele Priscila.pdf: 2574355 bytes, checksum: 6ecd817b9f806a21743e5671cf9f96c5 (MD5) Previous issue date: 2013-02-25 / Cutaneous aging is a complex biological phenomenon that depends on a number of intrinsic, responsible for chronological aging and the extrinsic factors, which is responsible for cutaneous photo aging. Another important factor that must be taken into account is the appearance of wrinkles caused by repeated movements of the facial muscles. Currently there is a huge demand for anti-aging products and one of the strategies that is most commonly employed for this end, is the application of botulinum toxin Type A which is regarded as the principal treatment for the mitigation of wrinkles. Another active substance which functions in the same way as botulinum toxin A, is acetyl hexapeptide-3, which is marketed under the name of Argireline. This anti-aging agent acts in a similar way to botulinum toxin by smoothing out the expressive lines and wrinkles caused by repeated movements. Both the active agents are responsible for the release of acetylcholine at the neuromuscular junction. One of the main differences between these active agents is in their form of application since, whereas botulinum toxin A is administered by injection, acetyl hexapeptide-3 is used in the form of a cream for topical application. Given the fact that the two active agents are hydrophilic neuropeptides of high molecular mass, their ability to penetrate the skin is improbable. Thus, the purpose of this study was to develop, characterize and evaluate systems comprising nanocarriers that are able to allow or improve the permeation of these active agents through the skin. Acetyl hexapeptide-3 was encapsulated in liposomes and botulinum toxin was encapsulated through two systems, liposomes and polymeric nanoparticles. The evaluation of the acetyl-hexapeptide-3 showed an encapsulation efficiency of 95%. The vesicles showed a zeta potential of -31 mV. The results demonstrated that the active agent had a good cutaneous permeation and its encapsulation did not affect the permeation through the skin. The botulinum toxin Type A were successfully encapsulated into nanoparticles by multiple emulsion method and solvent evaporation. The nanoparticles had an average size of approximately 570 nm and a zeta potential of -7,40 mV, which is characteristic of the polyester used. The multilamellar liposomes were obtained through a method that involved hydrating the lipid film and showed an average size of approximately 1370 nm and a zeta potential of -37 mV. It was possible to demonstrate by means of infrared fourier transform spectroscopy, the presence of botulinum toxin Type A in the polymeric nanoparticles and the liposomes. The in vitro assays that were carried out suggested that the liposomes that contained the botulinum toxin Type A had an effect on the neurotransmitters in the assays that were conducted, whereas it was not possible to identify the same effect in the nanoparticles that contained the botulinum toxin.These results showed that the encapsulation of botulinum toxin A can be an alternative for the topical application of botulinum toxin Type A in a non-invasive way and with a good deal of comfort to the patient. With regard to acetyl hexapeptide-3, it can be claimed that this active agent when released shows the same degree of penetration through the skin as the encapsulated active agent. This suggests that the penetration of acetyl hexapeptide-3 does not undergo any alteration when encapsulated in liposomes. Thus, the active agent can be applied topically in this way. / O envelhecimento cutâneo é um fenômeno biológico complexo que depende da combinação de fatores intrínsecos, responsáveis pelo envelhecimento cronológico e extrínsecos, responsáveis pelo fotoenvelhecimento cutâneo. Outro fator importante a ser considerado é o aparecimento de rugas causadas pelos movimentos faciais repetitivos. Atualmente é grande a procura por produtos antienvelhecimento e, uma das estratégias mais empregada para esse fim é a aplicação da toxina botulínica do tipo A, que é considerada a principal atenuante de rugas. Outro ativo empregado com a mesma função da toxina botulínica é o acetil hexapeptídeo-3, comercialmente conhecido como Argireline. Este agente antienvelhecimento age de forma similar à toxina botulínica, reduzindo as linhas e rugas de expressão, ambos atuam na liberação da acetilcolina na junção neuromuscular. Uma das principais diferenças entre esses ativos está em sua forma de aplicação, enquanto a toxina botulínica é aplicada por meio de injeções intramusculares, o acetil hexapeptídeo-3 é empregado na forma de creme para aplicação tópica. Considerando que os dois ativos são neuropeptídios hidrofílicos de elevada massa molar, sua penetração através da pele é improvável. Assim, o objetivo deste trabalho foi desenvolver, caracterizar e avaliar sistemas constituídos por nanocarreadores capazes de permitir ou melhorar a permeação destes ativos através da pele. O acetil hexapeptídeo-3 foi encapsulado em lipossomas e a toxina botulínica foi encapsulada em dois sistemas, lipossomas e nanopartículas poliméricas. A avaliação do acetil exapeptídeo-3 mostrou uma eficiência de encapsulação de 95%. As vesículas apresentaram um potencial zeta de -31 mV. Os resultados demonstraram que o ativo apresentou boa permeação cutânea e sua encapsulação não afetou a permeação através da pele. A toxina botulínica foi encapsulada com sucesso em nanopartículas poliméricas, pelo método de emulsão múltipla e evaporação do solvente. As nanopartículas apresentaram tamanho médio de aproximadamente 570 nm e potencial zeta de –7,40 mV, característico do poliéster utilizado. Os lipossomas multilamelares obtidos por hidratação do filme lipídico apresentaram diâmetro médio de aproximadamente 1370 nm e potencial zeta de -37mV. Foi possível demonstrar, por meio dos espectros de infravermelho com Transformada de Fourier, a presença da toxina botulínica tipo A nas nanopartículas poliméricas e nos lipossomas. Os ensaios realizados in vitro indicaram que os lipossomas contendo a toxina botulínica tipo A apresentaram efeito sobre os neurotransmissores nos ensaios realizados, entretanto não foi possível identificar o mesmo efeito nas nanopartículas contendo a toxina botulínica. Esses resultados mostraram que a encapsulação da toxina botulínica A pode ser uma alternativa para sua aplicação tópica, de forma não invasiva e com muito conforto ao paciente. Quanto ao acetil hexapeptídeo-3, pôde-se constatar que este ativo na forma livre apresentou a mesma penetração através da pele do que o ativo encapsulado, o que indica que a penetração do acetil hexapeptídeo-3 não sofre alteração quando encapsulado em lipossomas. Desta forma o ativo pode ser aplicado de forma direta.

Spinal cord injury (SCI) remains a significant cause for morbidity and mortality in North America. Bladder dysfunction following SCI is very common and could lead to severe complications including renal failure and autonomic dysreflexia (AD). AD involves life threatening episodes of hypertension in patients with SCI above T6 level. Current management protocols for AD are symptomatic and usually ineffective. Botulinum toxin-A (BTX-A), has been successfully used recently in SCI patients because it reduces the detrusor contractility via inhibiting acetylcholine release from efferent nerve endings. Recent evidence, however, suggests a sensory involvement via modulation of sensory neuropeptides, neurotransmitters, and receptors. It is still, however, unclear whether BTX-A can affect putative spinal neurons involved in AD. In this study we demonstrated that intravesical BTX-A treatment has blocked AD in rats with T4-SCI, and also provided a novel mechanism for the control of autonomic dysreflexia via a minimally invasive treatment modality.

Spinal cord injury (SCI) remains a significant cause for morbidity and mortality in North America. Bladder dysfunction following SCI is very common and could lead to severe complications including renal failure and autonomic dysreflexia (AD). AD involves life threatening episodes of hypertension in patients with SCI above T6 level. Current management protocols for AD are symptomatic and usually ineffective. Botulinum toxin-A (BTX-A), has been successfully used recently in SCI patients because it reduces the detrusor contractility via inhibiting acetylcholine release from efferent nerve endings. Recent evidence, however, suggests a sensory involvement via modulation of sensory neuropeptides, neurotransmitters, and receptors. It is still, however, unclear whether BTX-A can affect putative spinal neurons involved in AD. In this study we demonstrated that intravesical BTX-A treatment has blocked AD in rats with T4-SCI, and also provided a novel mechanism for the control of autonomic dysreflexia via a minimally invasive treatment modality.

No description available.

Formål: At undersøge, hvordan forældre til børn med spastisk cerebral pare­se (CP) oplevede effekt af behandling med Botulinumtoxin type A (BTX) på deres barns funktioner. Metode og materiale: Der blev anvendt triangulering af metode, kilder og observatører. Den kvali­ta­tive del bestod af to fokusgruppeinterview af eksperter og tre forælderinterview. Analyseme­toden var indholdsanalyse. Den kvantitative del bestod af en spørgeskemaundersøgelse til forældre til børn behandlet med BTX på Hillerød Hospital. Spørgeskemaer blev sendt til begge forældre til 47 børn. Resultat: Fokusgruppeinterviewene gav indblik i, at der i spørgeskemaet skulle spørges detaljeret til alle funktioner i barnets hverdag fra BTX blev tilbudt til efter endt effekt af BTX-behandling. Forældreinterviewene viste, at to af tre forældre ville tage imod et nyt tilbud om BTX-behandling; at ventetid på BTX-dagen var uacceptabel lang; at arbejdsgange på BTX-dagen burde optimeres, så de passede til handicappede børn. Spørgeskemaet blev returneret af 41 forældre til ialt 30 børn (to – 17 år). Børnene var behandlet med BTX på Hillerød Hospital i perioden 2004-2007. Af de 41 forældre var der 12 fædre og 29 mødre. Størst effekt af BTX oplevede forældrene på nedsættelse af barnets spasticitet. To tredjedele af de børn, som kunne gå før BTX behandling, fik forbedret gang. Derud­over sås effekt i nye måder at bevæge sig på. Toogtyve af 41 forældre så den effekt af BTX, som de havde fået stillet i udsigt. Størst effekt af BTX sås indenfor tre til seks måneder. Virkningen af BTX for det enkelte barn var forskellig fra behandling til behandling med en dalende tendens. Studiet viste, at forældre og børn blev godt mødt og blev hørt på børneafdelin­gen, var trygge ved persona­let, og oplevede BTX-dagen som god. Ventetid og et manglende måltid mad efter BTX var et stort pro­blem. Der sås ingen forskel i fædres og mødres opfattelse af sundhedsydel­ser til børn med CP. Nationale retningslinier for behandling med BTX ef­terspørges. Konklusion: Majoriteten af forældre ville tage mod et nyt tilbud om behandling med BTX. De væ­sentligste begrundelser var, at forældrene havde set god effekt af behandlingen, eller at de håbede på en effekt. Et mindretal af forældre ville ikke tage imod et nyt tilbud om behandling med BTX, fordi de enten ikke havde set effekt af behandlingen, eller fordi de oplevede mange strabadser for et meget lille udbytte. Praksis omkring BTX dagen bør udvikles og forbedres i forhold til handicappede børn / This study aimed to assess how parents of children with spastic Cerebral Palsy (CP) evaluated the efficacy of Botulinum Toxin A (BTX) treatment on their child. Method: Triangulation of measurement processes, i.e., qualitative assessment including two focus group interviews with experts and three interviews with parents; quantitative assessment involving questionnaires send to parents of 47 children treated with BTX at Hilleroed Hospital. The qualitative data were analysed using content analysis. Results: Focus group interviews indicated that questionnaires should seek detailed information about the daily activities of children undergoing BTX treatment, beginning treatment initiation and continuing until BTX effect declined. Parent interviews showed that two out of three would allow another BTX injection; waiting time on treatment day was too long; and hospital routines should be optimized to meet the needs of disabled children. Forty-one parents (12 fathers and 29 mothers) completed the questionnaire. The study included 30 children receiving BTX between 2004 and 2007. Parents described the best effect of BTX as reduced spasticity. Walking ability improved in 67% of children who could walk before treatment, and improved motor skills provided new ways of moving. Twenty-two parents believed that BTX treatment fulfilled doctors’ expectations. Maximum BTX effect occurred after three to six months. Effects differed with each treatment, mostly on a declining scale. Parents felt very welcome in the children’s ward and also felt that the staff listened to them; their overall assessment of treatment day experience was good. Major concerns included waiting time and a missing meal following treatment. Views of healthcare services for children with CP did not differ between fathers and mothers. The study indicates that development of national guidelines for BTX treatment will be highly recommended. Conclusion: The majority of parents agreed to repeat BTX injections, mainly because outcome was positive or met their expectations. A minority would refuse future BTX treatment, primarily due to lack of effect or because of significant problems for small benefit. Optimizing hospital routines on treatment day would help disabled children navigate BTX treatment more successfully / <p>ISBN 978-91-85721-52-8</p>