For the past ten 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 2024 CDKL5 Forum that took place in October 28-29 in Boston
La décima edición del Foro CDKL5 tuvo lugar en Boston, los días 28 y 29 de octubre de 2024. 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 2024.
Patients with Dravet syndrome caused by missense mutations in SCN1A are candidates for the clinical trials with ASOs and gene therapies to increase SCN1A expression, and will be candidate for this therapies after approval.
These are my main learnings from the Syngap Conference 2023 celebrated in Orlando the day before the American Epilepsy Society meeting.
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.
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. AES 2019 was the year of genetic therapies for the developmental and epileptic encephalopathies. This article highlights what I found the most interesting at the AES 2019 meeting.
La quinta edición del Foro CDKL5 tuvo lugar en Boston, los días 4 y 5 de noviembre. El Foro es una reunión anual exclusivamente por invitación 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 ha sido mi tercer Foro CDKL5, y el segundo desde que me uní a la Fundación Loulou.
Este es un repaso para los grupos de pacientes de las principales novedades del Foro CDKL5 2019. [SPANISH VERSION - ALSO AVAILABLE IN ENGLISH]
For the past five 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. This was the third Forum I attended, and my second since joining the Loulou Foundation.
Here are the main news and take-home messages from the 2019 CDKL5 Forum that took place in Boston in November 4 and 5.
The 2019 Dravet Syndrome Pipeline and Opportunities Review provides a review and analysis of 12 drug candidates in development for the treatment of Dravet syndrome, including 11 products that have received orphan drug designations. The Report includes the most recent updates on programs from GW Pharmaceuticals (Epidiolex / Epidyolex), Zogenix (Fintepla, ZX008), Biocodex (stiripentol) , Ovid Therapeutics (Soticlestat, OV935, TAK-935), Takeda Pharmaceutical, Supernus Pharmaceuticals (SPN-817, Huperzine), Xeris Pharmaceutical (diazepam), Epygenix Therapeutics (EPK-100, -200 and -300), NeuroCycle Therapeutics (NCT10015), PTC Therapeutics (ataluren), Stoke Therapeutics (STK-001), Encoded Therapeutics and OPKO Health (OPK88001, CUR-1915).
I used to say that at the patient communities “we set the agenda”. It turns out we didn’t, we were borrowing the agenda from scientific meetings. The 2019 CDKL5 Alliance International Research and Family Conference redefined what a patient-centered conference truly is. In this article I summarise the elements that make a meeting truly patient-centered.
There are currently 4 clinical trials ongoing or about to start in CDKL5 Deficiency Disorder: ataluren, ganaxolone, TAK-935 and fenfluramine. This article is a summary of where we are with clinical trials for CDKL5 Deficiency Disorder for families and other interested readers including what we know about these four drugs, their efficacy, at which level of clinical development they are at, and where can you learn more about these trials.
