Dravet Syndrome News

August 4, 2020August 4, 2020

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 (cannabidiol) indications to include patients ages 1 and older, according to its developer, GW Pharmaceuticals (GW). ... Read more

July 28, 2020July 28, 2020

Encoded Therapeutics Raises $135M to Advance Potential Dravet Gene Therapy

Dravet Syndrome News

Encoded Therapeutics has raised $135 million to support the first human clinical trials for ETX101, its gene therapy for SCN1A-positive Dravet syndrome patients. The funding also will be used for a natural ... Read more

July 21, 2020July 21, 2020

Epidiolex May Interact With Diacomit But With Depacon Seems OK, Small Trial Finds

Dravet Syndrome News

Use of cannabidiol-based Epidiolex with two anti-epileptic treatments do not appear to raise major safety concerns, with a small rise in blood concentrations of Diacomit (stiripentol) and no changes of clinical importance with Depacon ... Read more

July 14, 2020July 14, 2020

Fintepla Approval ‘Fills Us With a Lot of Pride,’ Zogenix CEO Says in Interview

Dravet Syndrome News

The recent approval of Fintepla (fenfluramine) was a long time coming for its developer, Zogenix, and an event that CEO Stephen Farr, PhD, describes as a “very proud moment for ... Read more

July 7, 2020July 7, 2020

UK Eases Restrictions on Epidyolex for Treating Epileptic Disorders

Dravet Syndrome News

The United Kingdom Home Office has changed the classification of GW Pharmaceuticals‘ Epidyolex — a treatment for epileptic conditions including Dravet syndrome — from a Schedule 2 drug to a Schedule 5 drug. ... Read more

June 30, 2020July 1, 2020

Enrollment Opens in MONARCH Trial Testing STK-001 in Young Patients

Dravet Syndrome News

Stoke Therapeutics has begun enrollment for its Phase 1/2a MONARCH study, investigating the safety, tolerability, and effectiveness of STK-001 in children and adolescents with Dravet syndrome. The MONARCH trial (NCT04442295) ... Read more

June 26, 2020June 26, 2020

Fintepla Earns FDA Approval for Treatment of Seizures in Dravet Syndrome

Dravet Syndrome News

Note: This story was updated June 26, 2020, to note that the high dose of Fintepla used in the Phase 3 trials was 0.7 mg/kg/day rather than 0.8, as well ... Read more

June 23, 2020July 24, 2020

Alexion Charitable Foundation Giving $1.1M to Assist Rare Disease Community During Pandemic

Dravet Syndrome News

The Alexion Charitable Foundation has awarded $1.1 million in grants to programs that support those with rare diseases during the COVID-19 pandemic, the organization recently announced. The grants will support ... Read more

June 23, 2020June 23, 2020

Epilepsy Surgery May Not Be Advised in Dravet With SCN1A Mutations, Study Finds

Dravet Syndrome News

Epilepsy surgery may be ineffective, and even detrimental, in people with Dravet syndrome caused by mutations in the SCN1A gene, a small multicenter study suggests. While patients with other SCN1A-associated ... Read more

June 16, 2020June 16, 2020

CBD May Have Its Own Anti-epileptic Properties, Regardless of Clobazam Use

Dravet Syndrome News

Add-on treatment with Epidiolex — an oral form of cannabidiol (CBD) — effectively drops the frequency of seizures in people with Dravet syndrome and Lennox‑Gastaut syndrome (LGS), regardless of co-administration ... Read more

June 10, 2020July 11, 2020

Coalition Will Address Racial Disparities in Rare Disease Communities

Dravet Syndrome News

The Black Women’s Health Imperative (BWHI) recently created a Rare Disease Diversity Coalition focused on reducing racial disparities in the rare disease community. Getting a timely and accurate diagnosis for ... Read more

June 9, 2020June 10, 2020

‘Shine Forward with Dravet’ Is Online Support for Families of Patients

Dravet Syndrome News

Pharmaceutical company Zogenix has launched “Shine Forward with Dravet,” an online resource to support the parents and siblings of children with Dravet syndrome. The new resource, which can be accessed ... Read more

June 2, 2020June 2, 2020

Dravet Syndrome Awareness Month is Here … (Online)

Dravet Syndrome News

In light of the COVID-19 pandemic, there are several ways to get involved virtually to support Dravet Syndrome Awareness Month, observed each June. Organized by the Dravet Syndrome Foundation (DSF), ... Read more

May 28, 2020June 28, 2020

COVID-19 Delaying Rare Disease and Gene Therapy Trials, Pharma Execs Say

COVID-19 News, Dravet Syndrome News

While the ongoing COVID-19 pandemic won’t have much of an impact on cash available for new biotech startups, it has begun to cause delays in the development of gene therapies ... Read more

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