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Novedades en Dravet durante el segundo día del congreso EILAT XVI

Volvemos hoy con un resumen de lo aprendido durante el segundo día del congreso EILAT XVI.

En el día de hoy, Brian Klein, Director del Programa de Cribado de Terapias para la Epilepsia (ETSP, por sus siglas en inglés) en la División de Investigación Traslacional del NINDS (el Instituto Nacional de Trastornos Neurológicos y Accidentes Cerebrovasculares de los Estados Unidos), habló delante de la audiencia internacional del EILAT XVI sobre uno de nuestros modelos de ratón del síndrome de Dravet que porta una mutación en SCN1A, la A1783V, encontrada en algunos pacientes. Nuestro ratón es el único modelo animal del síndrome de Dravet incluido en este programa de cribado, siendo utilizado por investigadores de todo el mundo para probar la eficacia y seguridad preclínica de potenciales terapias novedosas para el síndrome de Dravet, tanto fármacos tradicionales como terapias modificadoras de la enfermedad. Recordaréis que el año pasado os hablamos de este programa y de cómo los científicos involucrados en el cribado han probado la valía de nuestro modelo de ratón para el estudio de nuevas terapias para el síndrome de Dravet. Podéis volver a leer el artículo AQUÍ.

También hemos conocido datos adicionales sobre el ácido valproico, el cual sabéis que sigue siendo el fármaco por excelencia cuando se trata por primera vez a un niño con síndrome de Dravet. Dan Abrams, del equipo ejecutivo  de Cerebral Therapeutics, mostraba resultados prometedores tras la administración intracerebroventricular de valproato a cinco pacientes con crisis focales durante un ensayo clínico. Este tipo de administración favorece la infusión continua del fármaco en la región concreta del cerebro donde la crisis epiléptica se está generando, y parece que en el caso del valproato se ha conseguido una reducción media de las crisis del 60 al 80%, incluso tras cuatro años de tratamiento y con muchos pacientes libres de crisis por periodos largos de tiempo. ¿Podría el valproato intracerebroventricular ser también efectivo en pacientes con síndrome de Dravet y, también importante, podría este sistema de administración usarse con otros fármacos ya aprobados para el síndrome de Dravet? Estaremos pendientes en el futuro de posibles datos en este sentido.

Farhad Sahebkar, director médico senior en Jazz Pharmaceuticals, además de repasar los resultados ya conocidos por todos sobre la eficacia del cannabidiol en el tratamiento del síndrome de Dravet —que podéis recordar en nuestro resumen anual de fármacos en camino de fin de 2021—, comentó que están estudiando formas de cannabidiol con un mayor perfil de seguridad para el paciente así como de biodisponibilidad, con la intención de que el fármaco actúe sobre el organismo de una forma más eficaz. Jazz plantea, por tanto, la posibilidad de producir Epidyolex en cápsulas en lugar de como solución oral.

Mélanie Langlois, directora de investigación clínica en Zogenix (ahora parte de UCB), nos habló de la Fenfluramina (Fintepla), de la que podéis profundizar en nuestro resumen anual de fármacos en camino de fin de 2021. Durante la ronda de preguntas, se puso de manifiesto que hay que seguir trabajando para conseguir reducir cada vez más las crisis, ya que se ha visto que no es igual de efectivo en todos los pacientes. Mélanie Langlois expresa que desde Zogenix van a seguir trabajando para que los resultados sean cada vez mejores.

Elaine Wirrell, directora del Departamento de Epilepsia Pediátrica en la Clínica Mayo de Rochester, Minnesota, y miembro del comité asesor de la Fundación Dravet en E.E.U.U., revisó datos sobre el estiripentol, medicamento del cual los adultos también pueden beneficiarse, aunque tienen un elevado riesgo de presentar hiperamonemia. Como sabéis, se trata de un tratamiento que reduce las crisis convulsivas al combinarse con valproato y clobazam, y que podéis revisar en nuestro resumen anual de fármacos en camino o en el webinar que realizamos sobre él.

Por la tarde, las distintas charlas se enfocaron a distintas vías de administración de terapias de rescate, como el Diazepam o el Alprazolam.

Michael A. Rogawski, profesor de Farmacología y Neurología de la Universidad de California, y asesor científico de Acquestive Therapeutics, propuso la administración del Diazepam mediante láminas de film bucal, que se colocarían en la parte interior de la mejilla, para el tratamiento de las convulsiones. De momento, esta forma de administración se encuentra en ensayos clínicos fase 1 en EE.UU., comparando su farmacocinética con otras formas de administración existentes.

Por su parte, Sunita Misra, directora médica senior en Neurelis y con la que tuvimos la ocasión de comer ayer, confirmó la seguridad y eficacia del spray nasal de Diazepam, llamado Valtoco®, ya aprobado para su uso de emergencia en E.E.U.U. en pacientes de 6 años de edad o más. En la actualidad se está llevando a cabo un estudio para el uso de esta nueva forma de administración de Diazepam en pacientes más jóvenes.

Por último, Robert Roebling, director médico senior de UCB, nos ha presentó el dispositivo de alprazolam inhalado llamado Staccato®. El alprazolam es un medicamento de rescate similar al diazepam que ayuda a frenar las crisis. Aún se encuentra en fase de investigación, ya que no es recomendable para todos los pacientes ni para todos los tipos de crisis epilépticas.

Las sesiones de mañana estarán destinadas a los dispositivos médicos para el tratamiento y manejo de la epilepsia. ¡Os seguiremos informando!

Un sueño, una meta.

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Participamos en la Conferencia Europea de Enfermedades Raras y Productos Huérfanos 2022, donde además nuestro Presidente, José Ángel Aibar, ha presentado el póster “Determinación de lo que representa valor en el tratamiento del Síndrome de #Dravet desde la perspectiva de los pacientes españoles mediante la Metodología del Análisis de Decisión Multicriterio”.

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¿Te has descargado ya tu Dorsal Solidario 2022?

TERMS & CONDITIONS

The European Dravet Syndrome Advanced Therapies (EDSAT) Meeting 2022 is organised by Dravet Syndrome Foundation Spain, whose registered office is located at Calle Doctor Fleming, 30, 28036 Madrid, Spain.

The conference shall take place at the Illustrious Official College of Physicians of Madrid, at Calle de Santa Isabel, 51, 28012 Madrid, Spain, on Friday 1st of April 2022. Attendance to this conference is free of charge, but mandatory for organisational reasons. Participants must register online at www.dravetconference.com. Upon registration, each participant will receive an electronic confirmation by email. On-site registration will not be be possible. If a participant has not received the official email registration confirmation within 3 days, they must contact the conference secretariat at research@dravetfoundation.eu.

The conference registration includes: Attendance to all sessions, oral and visual presentations, all coffee and lunch breaks, and conference documentation with all information about the event. The registration does not comprise travelling and accommodation costs or any other personal expenses.

Admittance to the workshop without registration cannot be granted.

Conference Attendance
Upon arrival at the Illustrious Official College of Physicians of Madrid the day of the event, all participants have to register at the onsite Conference Registration Desk. The desk will open on 1st of April at 8:30 a.m. Upon registration onsite, the conference team will provide participants with information about the event and a badge, which should be worn visibly at all times. The personalised badge authorises participants to attend all conference sessions, and lunch and coffee breaks.

Cancellation Policy
Dravet Syndrome Foundation Spain reserves the right to cancel any registration forthwith and without liability on its part in the event of any damage to, or destruction of the venue by fire, shortage of labour, strikes, industrial unrest, public health measures, or any other cause beyond the control of the Dravet Syndrome Foundation Spain, which shall prevent it from performing its obligations. In these circumstances, every effort will be made to find an alternative format/venue or propose a new date for the conference. Should the conference be cancelled for any reason, airfares, hotels, or other costs incurred by the participants are not reimbursed by the Dravet Syndrome Foundation Spain.

Any participant is allowed to cancel his/her registration but must notify this to the conference organisers by e-mail at research@dravetfoundation.eu within a minimum of 10 days
prior the event. Failure to attend the event without prior written notification
by email may incur a no-show fee, which shall cover individual organizational,
material and catering costs.

Name Change
All registrations are only valid for the respective person mentioned in the form. A registered participant unable to attend the conference may nominate (free of charge) a substitute participant by notifying the conference organisers by email at research@dravetfoundation.eu before 24th of March 2022. The procedure allows giving the last name, first name, and email address of the new participant.

Personal Data
Upon registration, the participant authorises Dravet Syndrome Foundation Spain to use their personal data stated above to inform the participant of any further conferences, events, or information concerning the conference and Dravet Syndrome Foundation Spain’s activities. The conference booklet online will only contain authors, titles, and affiliations of submitted abstracts, but no full abstract texts. The conference printed booklet will contain full abstract texts from all participants. Conference certificates will be sent to the participants as a PDF document upon request at research@dravetfoundation.eu. Only the name & affiliation will be stated in the certificate, not the email addresses of the participants.

Information is collected by an automated computer system that manages registrations. Security and confidentiality are guaranteed by state-of-the-art technical tools. However, Dravet Syndrome Foundation Spain declines any responsibility concerning data security transmitted through the internet.

Photography
The conference is held in public; therefore, we do not ban participants, exhibitors, partners and other companies from taking photographs of conference activities and participants, and share it via social media. By participating in the conference, you approve that general pictures will be taken and used for our Social Media output, our websites and other communication purposes. You hereby waive any right to oppose, and expressly authorise the organiser to make them available on the Internet or as it may seem fit. Author’s presentation slides shall not be photographed by any conference participant.

Disclaimer
The conference organiser cannot be held responsible for any loss, injury or damage to any property, whatever the cause may be. Should, for any reason outside the organiser’s control (e.g., political or economic circumstances or a case of ‘force majeure’), the venue or speakers change, or the event be cancelled, the organiser will endeavour to reschedule, but shall not be held responsible for any costs, damages or expenses incurred by the participants. If for any reason the organiser decides to make material changes to this conference, the organiser is not responsible for airfares, hotels or other costs incurred by the participants. The participant takes part in the conference, all tours, and trips at his own risk.

Copyright
The participant is allowed to use the conference documentation exclusively for his/her personal needs.

TERMS & CONDITIONS

The Dravet Syndrome Conference 2022 is organised by Dravet Syndrome Foundation Spain, whose registered office is located at Calle Doctor Fleming, 30, 28036 Madrid, Spain.

The conference shall take place at the Illustrious Official College of Physicians of Madrid, at Calle de Santa Isabel, 51, 28012 Madrid, Spain, on Thursday 31st of March 2022. Attendance to this conference is free of charge, but mandatory for organisational reasons. Participants must register online at www.dravetconference.com. Upon registration, each participant will receive an electronic confirmation by email. On-site registration will not be be possible. If a participant has not received the official email registration confirmation within 3 days, they must contact the conference secretariat at research@dravetfoundation.eu.

The conference registration includes: Attendance to all sessions, oral and visual presentations, all coffee and lunch breaks, and conference documentation with all information about the event. The registration does not include travel and accommodation costs or any other personal expenses.

Admittance to the conference without registration cannot be granted.

Conference Attendance
Upon arrival at the Illustrious Official College of Physicians of Madrid the day of the event, all participants have to register at the onsite Conference Registration Desk. The desk will open on 31st of March at 8:30 a.m. Upon registration onsite, the conference team will provide participants with information about the event and a badge, which should be worn visibly at all times. The personalised badge authorises participants to attend all conference sessions, and lunch and coffee breaks. 

Cancellation Policy
Dravet Syndrome Foundation Spain reserves the right to cancel any registration forthwith and without liability on its part in the event of any damage to, or destruction of the venue by fire, shortage of labour, strikes, industrial unrest, public health measures, or any other cause beyond the control of the Dravet Syndrome Foundation Spain, which shall prevent it from performing its obligations. In these circumstances, every effort will be made to find an alternative format/venue or propose a new date for the conference. Should the conference be cancelled for any reason, airfares, hotels, or other costs incurred by the participants are not reimbursed by the Dravet Syndrome Foundation Spain.

Any participant is allowed to cancel his/her registration but must notify this to the conference organisers by e-mail at research@dravetfoundation.eu within a minimum of 10 days prior the event. Failure to attend the event without prior written notification by email may incur a no-show fee, which shall cover individual organizational, material and catering costs.

Name Change
All registrations are only valid for the respective person mentioned in the form. A registered participant unable to attend the conference may nominate (free of charge) a substitute participant by notifying the conference organisers by email at research@dravetfoundation.eu before 24th of March 2022. The procedure allows giving the last name, first name, and email address of the new participant.

Personal Data
Upon registration, the participant authorises Dravet Syndrome Foundation Spain to use their personal data stated above to inform the participant of any further conferences, events, or information concerning the conference and Dravet Syndrome Foundation Spain’s activities. The conference booklet online will only contain authors, titles, and affiliations of submitted abstracts, but no full abstract texts. The conference printed booklet will contain full abstract texts from all participants. Conference certificates will be sent to the participants as a PDF document upon request at research@dravetfoundation.eu. Only the name & affiliation will be stated in the certificate, not the email addresses of the participants.

Information is collected by an automated computer system that manages registrations. Security and confidentiality are guaranteed by state-of-the-art technical tools. However, Dravet Syndrome Foundation Spain declines any responsibility concerning data security transmitted through the internet.

Photography
The conference is held in public; therefore, we do not ban participants, exhibitors, partners and other companies from taking photographs of conference activities and participants, and share it via social media. By participating in the conference, you approve that general pictures will be taken and used for our Social Media output, our websites and other communication purposes. You hereby waive any right to oppose, and expressly authorise the organiser to make them available on the Internet or as it may seem fit. Author’s presentation slides shall not be photographed by any conference participant. 

Disclaimer
The conference organiser cannot be held responsible for any loss, injury or damage to any property, whatever the cause may be. Should, for any reason outside the organiser’s control (e.g., political or economic circumstances or a case of ‘force majeure’), the venue or speakers change, or the event be cancelled, the organiser will endeavour to reschedule, but shall not be held responsible for any costs, damages or expenses incurred by the participants. If for any reason the organiser decides to make material changes to this conference, the organiser is not responsible for airfares, hotels or other costs incurred by the participants. The participant takes part in the conference, all tours, and trips at his own risk.

Copyright
The participant is allowed to use the conference documentation exclusively for his/her personal needs.