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What I learnt at the SYNGAP Conference 2023

These are my main learnings from the Syngap Conference 2023 celebrated in Orlando the day before the American Epilepsy Society meeting.

What I learnt at the SYNGAP Conference 2023
Dracaena Consulting
TOP 5 INSIGHTS FROM THE AMERICAN EPILEPSY ...

The American Epilepsy Society (AES) meeting is the largest epilepsy meeting of the year, and because it takes place every month of December it also serves as an annual review on the understanding and treatment of epilepsies. These are my top 5 insights from the American Epilepsy Society 2023 meeting.

TOP 5 INSIGHTS FROM THE AMERICAN EPILEPSY SOCIETY MEETING 2023
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REPASO DEL FORO CDKL5 2023

La novena edición del Foro CDKL5 tuvo lugar en Boston, los días 6 y 7 de noviembre. El Foro es una reunión anual que organiza la Fundación Loulou y en la que científicos y miembros de la industria farmacéutica se reúnen con representantes de la comunidad de pacientes para repasar los últimos avances en el campo.

Este es un repaso para los grupos de pacientes de las principales novedades del Foro CDKL5 2023.

REPASO DEL FORO CDKL5 2023
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MAIN LESSONS FROM THE 2023 CDKL5 FORUM

For the past nine years the Loulou Foundation hosts an annual meeting where scientists and drug developers working on CDKL5 deficiency, together with representatives from patient organizations, meet to discuss the latest advances.

Here are the main news and take-home messages from the 2023 CDKL5 Forum that took place in November 6-7 2023

MAIN LESSONS FROM THE 2023 CDKL5 FORUM
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Dónde está la mutación de tu hijo en CDKL5, y ...

Una conversación que suele salir mucho en reuniones de familias CDKL5 es la posición de la mutación de sus hijos en el gen, por si eso nos ayuda a saber la severidad de la enfermedad cuando crezcan. Os resumo abajo un listado de publicaciones y sus conclusiones.

Dónde está la mutación de tu hijo en CDKL5, y qué nos dicen los gemelos

Orphan drug approvals: 2018 set new record for the EMA, but some red flags

Orphan drug approvals: 2018 set new record for the EMA, but some red flags

2018 saw a record year in orphan drug approvals in Europe, but there are reasons to worry. Year after year, the number of orphan drug approvals in Europe is only one fifth to one third of the number of drug approvals in the US. Also, if orphan designations represent an early marker of the orphan drug development trend, then we might expect a decrease in the number of approvals in the immediate future. This article reviews the number of orphan drug designations and approvals in Europe in the 2000-2018 period to understand the trends that might impact the number of orphan drug approvals in the next few years.

2018 in review: Dravet syndrome milestones

2018 in review: Dravet syndrome milestones

With 2018 now behind us, it is time to review how well companies working on Dravet syndrome delivered based on the timelines that they had announced at the beginning of the year. While it is hard to predict exactly when many milestones are going to happen, in particular those still over half a year away or more, the class of 2018 did quite well overall, and many of the news that we were expecting took place on schedule – with some exceptions.

Engaged for 20 years: an orphan drug designation from 1995 just got approved in 2018

Engaged for 20 years: an orphan drug designation from 1995 just got approved in 2018

The Food and Drug Administration (FDA) set a new record in 2018 with the highest number of new drug approvals in the last two decades. The FDA also set a new record in orphan drug approvals in 2018, granting 86 new marketing authorizations for drugs treating rare diseases. In this article I review the delay between orphan drug designation and orphan drug approval, and identify how in many cases orphan drugs wait 10 or more years after reviewing the orphan drug designation and before they get approved.

Top 5 insights from the American Epilepsy Society meeting (2018)

Top 5 insights from the American Epilepsy Society meeting (2018)

Every year the American Epilepsy Society (AES) meeting gets larger. This year, over 6,000 people gathered in New Orleans to discuss the latest information about epilepsy care and the development of new treatments for epilepsy. The 2018 meeting captured the latest developments in the field of epilepsy drug development, where rare disease populations and new technologies are two areas of considerable growth and that are changing the way we will treat epilepsy. This article highlights what I found the most interesting at the AES 2018 meeting.

IMPATIENT SERIES #5 – EVALUATING AND TRACKING PROJECTS

IMPATIENT SERIES #5 – EVALUATING AND TRACKING PROJECTS

When you let the scientific community know that your organization is open to fund research around your rare disease you are likely to get many research proposals from academic groups. These will come in different qualities, and will have different relevancefor your disease. This entry adds to Impatient Series I and to the ImpatientRevolution book and discusses in more detail how to determine that a proposed project is the right one and how to monitor its progress.

Main lessons from the 2018 CDKL5 Forum

Main lessons from the 2018 CDKL5 Forum

For the past four years the Loulou Foundation hosts an annual “by invitation only” meeting where scientists and drug developers working on CDKL5 deficiency, together with representatives from patient organizations, meet to discuss the latest advances. This was the second Forum I attended, and my first since joining the Loulou Foundation.

Here are the main news and take-home messages from the 2018 CDKL5 Forum that took place in London, UK, in October 22 and 23.