The Need for Awareness of SUDEP

By |2019-07-19T17:33:47-05:00July 19th, 2019|Categories: Decoding Dravet Blog|

You can anticipate that you will have some worries when you become a parent. You are fully responsible for this new little person and for keeping them safe. When they are home there are dozens of safety measures you can implement - smoke detectors, fencing, safety gates, etc. As they grow and develop, you [...]

Double Down Beantown

By |2019-07-15T16:37:45-05:00July 16th, 2019|Categories: Our Community|

2019 Double Down Beantown – Boston Bets Against Dravet Congratulations and thank you Karina & Daniel Fischer, Lauren & Jon Forker, Stephanie & Josh Goldman, Abby & Micah Hemani, Amy & Lenny Lopez, and Barbara & John Swoyer. These 6 Boston area families joined forces to host an amazing first time gala fundraiser [...]

5K for Dravet: Denver

By |2019-07-15T19:41:08-05:00July 16th, 2019|Categories: Events|

2019 Steps Toward a Cure: Denver, CO Join us for a fun, family-friendly event that includes a professioanl timed 5k run, 1 mile walk and family fun day at 5K for Dravet: Colorado on Sunday, August, 11, 2019 5K for Dravet: Colorado was started in 2013 by the Craner Family in honor of [...]

Patient Voice Webinar

By |2019-07-16T07:39:58-05:00July 16th, 2019|Categories: DSF Programs|

Patient Voice in the Development of Research Webinar Dravet Syndrome Foundation (DSF) has developed a webinar because there is an increasing recognition of the importance of the involvement of patients in the development of research that will impact their disease state. In 2018, DSF, received a generous grant from the Patient Centered Outcomes [...]

$104M to Gene Therapy

By |2019-07-15T20:31:01-05:00July 16th, 2019|Categories: Research / Treatments|

Encoded Therapeutics Finances $104 Million to Advance Lead Gene Therapy Program Encoded Therapeutics, Inc. announced they will use $104 million in Series C financing to advance its lead gene therapy program in Dravet syndrome and its preclinical pipeline. They also unveiled their precision gene therapy platform. Encoded presented preclinical data on their approach [...]

Fintepla RTF Update

By |2019-07-15T16:38:30-05:00July 16th, 2019|Categories: Research / Treatments|

Zogenix To Resubmit New Drug Application Zogenix, Inc. anticipates that they will be resubmitting their New Drug Application (NDA) to the U.S. Food & Drug Administration (FDA) in the third quarter, 2019 without additional chronic toxicity studies for Fintepla (ZX008, low dose fenfluramine). In April, the FDA issued a Refusal to File (RTF) [...]

NaV1.1 and NaV1.6 selective compounds reduce the behavior phenotype in a novel zebrafish model for Dravet Syndrome

By |2019-07-15T23:23:04-05:00July 14th, 2019|Categories: DSF Research Review|Tags: , , |

Dravet syndrome is caused by loss-of-function mutations in the SCN1A gene, which codes for the alpha subunit of the NaV1.1 sodium channel. However, there are other sodium channels, and it is known that gain-of-function mutations in SCN8A, the gene that codes for the alpha subunit of the NaV1.6 sodium channel, can lead to severe [...]

Engineering brain activity patterns by neuromodulator polytherapy for treatment of disorders

By |2019-07-15T23:15:28-05:00July 14th, 2019|Categories: DSF Research Review|Tags: , , |

This article is interesting for a couple of reasons. First, it describes the use of zebrafish as a model for Dravet syndrome. Zebrafish have become very useful in medical research. They are small, and easy and inexpensive to maintain and employ in drug response studies, especially in comparison to mice. Of course, although they [...]

The Efficacy of Ketogenic Diet in 60 Chinese Patients With Dravet Syndrome

By |2019-07-15T23:17:09-05:00July 13th, 2019|Categories: DSF Research Review|Tags: , , |

This was a retrospective study, gathering information on Dravet patients who, over the last nine years, had followed a strict ketogenic diet for at least 12 weeks. In total, there were 60 children identified. The duration of their ketogenic diets ranged from 12 weeks to 54 months, with a median duration of 44 weeks. [...]

The Importance of Natural History Studies

By |2019-07-02T20:57:58-05:00July 12th, 2019|Categories: Decoding Dravet Blog|

As a parent of a 19 year old son with Dravet syndrome, I recognize that the idea of giving up your time to participate in any type of research may sound overwhelming or next to impossible. Your days are already packed from morning to night with the constant care of your child, including therapies, [...]