Dravet Syndrome News

May 26, 2020May 26, 2020

Enrollment to Start Later This Year in MONARCH Trial Testing STK-001 for Dravet

Dravet Syndrome News

Stoke Therapeutics plans to begin enrollment later this year of Dravet syndrome patients for part A of its Phase 1/2a MONARCH trial to evaluate the investigational therapeutic STK-001, according to ... Read more

May 19, 2020May 19, 2020

Gene Therapy Shows Promise in Mouse Model of Dravet Syndrome

Dravet Syndrome News

A gene therapy designed to increase the production of a subunit of the defective sodium channel in Dravet syndrome prolonged the survival, lowered seizures, and partially corrected abnormal behaviors in ... Read more

May 12, 2020May 12, 2020

Body Cooling May Shorten Refractory Seizures in Dravet and Other Epilepsies

Dravet Syndrome News

Used in addition to standard treatments, therapeutic hypothermia — based on lowering the body’s temperature — can shorten the duration of long-lasting seizures in drug-resistant forms of epilepsy, including Dravet ... Read more

May 5, 2020May 5, 2020

Epidiolex Lowered Premature Mortality in Dravet Mice, Studies Find

Dravet Syndrome News

Epidiolex, the first plant-derived cannabidiol (CBD) medication to be approved by the U.S. Food and Drug Administration (FDA), can reduce premature mortality and alleviate several behavioral deficits in mouse models of ... Read more

April 28, 2020April 28, 2020

Heart Abnormalities Unlikely Behind Sudden Unexpected Deaths in Dravet, Study Says

Dravet Syndrome News

Heart abnormalities are unlikely to be the reason behind the high rate of Sudden Unexpected Death in Epilepsy (SUDEP) in people with Dravet syndrome, a new study suggests, though further ... Read more

April 22, 2020April 22, 2020

NORD Opens COVID-19 Financial Aid Program for Rare Disease Community

Dravet Syndrome News

The National Organization for Rare Disorders (NORD) has opened a financial assistance program for people in rare disease community who are affected by the COVID-19 pandemic in the U.S. Called ... Read more

April 21, 2020April 21, 2020

Artificial Intelligence May Speed up Epilepsy Diagnoses, Study Suggests

Dravet Syndrome News

A newly developed artificial intelligence (AI) system could help expedite the diagnosis of epileptic conditions such as Dravet syndrome. The AI system was described in a study, titled “A propositional ... Read more

April 14, 2020April 14, 2020

Fintepla’s Potential Dual Mechanism of Action May Explain Effectiveness at Reducing Seizures in Dravet Syndrome

Dravet Syndrome News

In addition to promoting the release of serotonin, a brain chemical typically associated with feelings of well-being and happiness, Fintepla (fenfluramine) also seems to control the activity of sigma-1 receptors found ... Read more

April 8, 2020May 10, 2020

NORD’s Advice on COVID-19: You’re Stronger Than You Think

COVID-19 News, Dravet Syndrome News

First, the bad news: If you’re one of the 30 million or so Americans with a rare disease, you probably have lower immunity to the novel coronavirus than most people. ... Read more

April 7, 2020April 7, 2020

Epidiolex Now Available in the US as a Non-controlled Substance

Dravet Syndrome News

In a move that will give Dravet syndrome patients easier treatment access, the U.S. Drug Enforcement Administration (DEA) has reclassified Epidiolex (formerly GWP42003-P) as a non-controlled substance. In what the agency called ... Read more

March 24, 2020March 24, 2020

Epidiolex Still Effective at Half the Highest Approved Dose for Reducing Frequency of Convulsive Seizures in Children with Dravet

Dravet Syndrome News

Epidiolex (cannabidiol), as an add on-therapy administered at either a low or high dose, leads to similar clinically relevant reductions in the frequency of convulsive seizures in children with treatment-resistant Dravet syndrome, ... Read more

March 20, 2020April 21, 2020

Global Rare Disease Group’s Focus: 1,000 New Therapies by 2027, Despite COVID-19

Dravet Syndrome News

Even with the coronavirus pandemic ravaging Europe and much of the world, patient advocate Lucia Monaco, PhD, of Italy remains confident that the Paris-based nonprofit she chairs will see the ... Read more

March 17, 2020March 21, 2020

ELEKTRA Trial Completes Enrollment to Test Soticlestat in Children With Dravet and LGS

Dravet Syndrome News

The ELEKTRA clinical trial assessing the investigational oral therapy soticlestat (OV935/TAK-935) in children with Dravet syndrome and Lennox-Gastaut syndrome (LGS) has completed patient enrollment, Ovid Therapeutics recently announced. “We completed enrollment ... Read more

March 10, 2020March 21, 2020

Antisense Oligonucleotide Treatment Delays Seizures in Dravet Animal Study

Dravet Syndrome News

An antisense oligonucleotide therapy that lowers the amount of Scn8a transcripts — RNA molecules used as a template for the production of proteins — delayed seizure onset and prolonged the lifespan of ... Read more

March 9, 2020March 21, 2020

Pediatric Rare Disease Therapies Increase, But Most Repurposed, Study Finds

Dravet Syndrome News

The number of treatments for children with rare diseases has grown over the past decade, according to a new study. However, despite the increase, nearly 7,000 rare diseases are still lacking ... Read more