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SCN1A Gene Activation Reduced Seizures, Improved Behavior in Dravet Mice
Activating the SCN1A gene, which is deficient in most patients with Dravet syndrome, reduced febrile ...
More from Dravet Syndrome News
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Epidiolex Now Available in U.S. for Dravet, LGS Patients Age 1 and Older
The U.S. Food and Drug Administration (FDA) has expanded the age range for all Epidiolex ... -
Encoded Therapeutics Raises $135M to Advance Potential Dravet Gene Therapy
Encoded Therapeutics has raised $135 million to support the first human clinical trials for ETX101, its ... -
Epidiolex May Interact With Diacomit But With Depacon Seems OK, Small Trial Finds
Use of cannabidiol-based Epidiolex with two anti-epileptic treatments do not appear to raise major safety concerns, ... -
Fintepla Approval ‘Fills Us With a Lot of Pride,’ Zogenix CEO Says in Interview
The recent approval of Fintepla (fenfluramine) was a long time coming for its developer, Zogenix, ... -
National Survey Seeks to Assess Full Social, Economic Burdens of Rare Diseases in US
The EveryLife Foundation for Rare Diseases has launched a nationwide National Burden of Rare Disease ... -
UK Eases Restrictions on Epidyolex for Treating Epileptic Disorders
The United Kingdom Home Office has changed the classification of GW Pharmaceuticals‘ Epidyolex — a treatment for ... -
Enrollment Opens in MONARCH Trial Testing STK-001 in Young Patients
Stoke Therapeutics has begun enrollment for its Phase 1/2a MONARCH study, investigating the safety, tolerability, ... -
Fintepla Earns FDA Approval for Treatment of Seizures in Dravet Syndrome
Note: This story was updated June 26, 2020, to note that the high dose of ... -
Alexion Charitable Foundation Giving $1.1M to Assist Rare Disease Community During Pandemic
The Alexion Charitable Foundation has awarded $1.1 million in grants to programs that support those ... -
Epilepsy Surgery May Not Be Advised in Dravet With SCN1A Mutations, Study Finds
Epilepsy surgery may be ineffective, and even detrimental, in people with Dravet syndrome caused by ... -
CBD May Have Its Own Anti-epileptic Properties, Regardless of Clobazam Use
Add-on treatment with Epidiolex — an oral form of cannabidiol (CBD) — effectively drops the ... -
Coalition Will Address Racial Disparities in Rare Disease Communities
The Black Women’s Health Imperative (BWHI) recently created a Rare Disease Diversity Coalition focused on ... -
‘Shine Forward with Dravet’ Is Online Support for Families of Patients
Pharmaceutical company Zogenix has launched “Shine Forward with Dravet,” an online resource to support the ... -
Dravet Syndrome Awareness Month is Here … (Online)
In light of the COVID-19 pandemic, there are several ways to get involved virtually to ... -
COVID-19 Delaying Rare Disease and Gene Therapy Trials, Pharma Execs Say
While the ongoing COVID-19 pandemic won’t have much of an impact on cash available for ... -
Enrollment to Start Later This Year in MONARCH Trial Testing STK-001 for Dravet
Stoke Therapeutics plans to begin enrollment later this year of Dravet syndrome patients for part ... -
Eurordis Urges Expansion of Newborn Screening Initiatives Across Europe
European authorities must step up efforts to screen babies for a multitude of genetic disorders, ... -
Video Games Connect Chronically Ill Children Isolated at Home, Hospital
Dara Riva always had a rule that her 10-year-old son could play video games only ... -
Unity and EU-wide Efforts Focus of Online Rare Disease Meeting
Eurordis, a Paris-based coalition of national rare disease associations across Europe, hosted its first all-virtual ... -
Gene Therapy Shows Promise in Mouse Model of Dravet Syndrome
A gene therapy designed to increase the production of a subunit of the defective sodium ... -
Body Cooling May Shorten Refractory Seizures in Dravet and Other Epilepsies
Used in addition to standard treatments, therapeutic hypothermia — based on lowering the body’s temperature ... -
Epidiolex Lowered Premature Mortality in Dravet Mice, Studies Find
Epidiolex, the first plant-derived cannabidiol (CBD) medication to be approved by the U.S. Food and Drug ... -
Heart Abnormalities Unlikely Behind Sudden Unexpected Deaths in Dravet, Study Says
Heart abnormalities are unlikely to be the reason behind the high rate of Sudden Unexpected ... -
NORD Opens COVID-19 Financial Aid Program for Rare Disease Community
The National Organization for Rare Disorders (NORD) has opened a financial assistance program for people ... -
Artificial Intelligence May Speed up Epilepsy Diagnoses, Study Suggests
A newly developed artificial intelligence (AI) system could help expedite the diagnosis of epileptic conditions ... -
Fintepla’s Potential Dual Mechanism of Action May Explain Effectiveness at Reducing Seizures in Dravet Syndrome
In addition to promoting the release of serotonin, a brain chemical typically associated with feelings ... -
NORD’s Advice on COVID-19: You’re Stronger Than You Think
First, the bad news: If you’re one of the 30 million or so Americans with ... -
Epidiolex Now Available in the US as a Non-controlled Substance
In a move that will give Dravet syndrome patients easier treatment access, the U.S. Drug Enforcement ... -
Epidiolex Still Effective at Half the Highest Approved Dose for Reducing Frequency of Convulsive Seizures in Children with Dravet
Epidiolex (cannabidiol), as an add on-therapy administered at either a low or high dose, leads ... -
Global Rare Disease Group’s Focus: 1,000 New Therapies by 2027, Despite COVID-19
Even with the coronavirus pandemic ravaging Europe and much of the world, patient advocate Lucia ... -
ELEKTRA Trial Completes Enrollment to Test Soticlestat in Children With Dravet and LGS
The ELEKTRA clinical trial assessing the investigational oral therapy soticlestat (OV935/TAK-935) in children with Dravet syndrome ... -
Antisense Oligonucleotide Treatment Delays Seizures in Dravet Animal Study
An antisense oligonucleotide therapy that lowers the amount of Scn8a transcripts — RNA molecules used as ... -
Pediatric Rare Disease Therapies Increase, But Most Repurposed, Study Finds
The number of treatments for children with rare diseases has grown over the past decade, according ... -
Video: BioNews’ Social Media Campaign Highlights #WhatMakesMeRare
In recognition of Rare Disease Day Feb. 29, BioNews Services launched a social media campaign ... -
Molecule Seen to Lessen Epileptic Activity, Improve Learning in Early Study
A new small molecule was seen to stimulate certain brain receptors, called NMDA receptors, and ... -
Elsevier Opens Books, Review Articles to Rare Disease Researchers Starting Feb. 29
Beginning on Feb. 29, Rare Disease Day, chapters from notable scientific books and clinical review articles ... -
FDA Extends Review of Fintepla for Dravet Syndrome; Response Expected in June
The U.S. Food and Drug Administration (FDA) extended by three months the review period for ... -
Advice on Starting Nonprofit Groups for Rare and Other Diseases Focus of NORD Webinar
Starting a 501(c)(3) tax-exempt nonprofit isn’t easy, but the National Organization for Rare Disorders gave ... -
Fintepla Reduces Seizure Frequency in Dravet Children Who Respond Poorly to Diacomit Combo Regimens, Phase 3 Trial Shows
Fintepla (fenfluramine) reduced the frequency of convulsive seizures in children and adolescents with Dravet syndrome ...