The COVID-19 pandemic has raised health concerns worldwide, but particularly in communities that may be at higher risk from illness and infection. To understand this better in the Dravet syndrome community, Dravet Syndrome UK (DSUK) performed a survey of families caring for an individual living with Dravet syndrome from June-July 2020 to assess the impacts […]
The impact of COVID-19 in Dravet Syndrome: a UK survey Read More »
Serotonin (5-HT) signaling has been a major focus of drug development targeting seizures in Dravet syndrome (DS) in recent years. For example, the community recently saw the FDA-approval of Fintepla (fenfluramine) as an adjunctive (add-on) therapy for DS. Fenfluramine had been a known anti-epileptic agent since the 1980’s, particularly utilized in Europe, but its marketing
Lorcaserin Enters Clinical Trials for Dravet Syndrome Read More »
Just last month, a previous research review detailed how Stoke Therapeutics developed their targeted augmentation of nuclear gene output (TANGO), utilizing anti-sense oligonucleotides (ASOs) to upregulate expression of several genes, including SCN1A, in human cells and mouse brain. ASOs are single-stranded RNAs that bind to target RNA sequences and have the potential to alter the
In this brief report published in June, Vezyroglou et al detail the results of epilepsy surgery for focal seizures in 8 patients carrying mutations in the SCN1A gene that were predicted to be causal. Three of the 8 patients had a clinical diagnosis of Dravet syndrome. The surgical procedures were successful in reducing or eliminating
FINTEPLA (fenfluramine) was recently approved by the FDA for the treatment of seizures in Dravet syndrome. The FDA required Zogenix, Inc to include a black box warning on the label because fenfluramine belongs to a class of drugs that affect a specific serotonin receptor (5-HT2B), and these types of drugs have previously been associated with valvular
In this paper, Dyment et al detail a new mouse for the study of Dravet syndrome modeled after a patient mutation. This particular mutation (H939R) does not result in the typical haploinsufficiency where Nav1.1 sodium channel levels are reduced, but rather appears to affect the function of one copy of the sodium channel. Consistent with
In late June 2020, Voskobiynyk et al shared their recent manuscript on bioRxiv (pre-print before peer-review) detailing a novel mouse model of Dravet syndrome that carries the same mutation in a non-coding region as a patient diagnosed with Dravet syndrome. “Non-coding” means that the mutation is not located in an area of the DNA that
Stoke Therapeutics published a paper in Nature Communications detailing the mechanism of action for their new TANGO (targeted augmentation of nuclear gene output) therapy that utilizes ASO (antisense oligonucleotide) technology. While the applications of this technology have been discussed in presentations and meetings, this is the first publication detailing how it was developed. Cells use
I often reflect on the amount of time spent traveling to and from the numerous doctor and therapy appointments with my son over the years. Negotiating my work schedule, coordinating school and therapy schedules, packing medical equipment and medicines, loading my son and his wheelchair into the car, making the sometimes-long drives to the clinic,
Sustaining Telehealth Beyond the COVID-Era Read More »
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