Dravet Syndrome News

October 20, 2020October 20, 2020

First Patient Enrolled in Phase 2 Trial of EPX-100, Potential Add-on Therapy for Dravet

The first patient has been enrolled in Epygenix Therapeutics’ multicenter Phase 2 clinical trial that will assess the effectiveness and safety of EPX-100 as an add-on therapy in children with ... Read more

October 13, 2020October 13, 2020

Eisai Begins Phase 3 Trial Testing Lorcaserin at Reducing Dravet Seizure Frequency

Dravet Syndrome News

Following consultation with the U.S. Food and Drug Administration (FDA), Eisai has initiated a Phase 3 clinical trial called MOMENTUM 1 to evaluate lorcaserin as a potential treatment for Dravet ... Read more

October 6, 2020October 6, 2020

Dravet Syndrome Foundation Readies for Online ‘Day of Dravet’

Dravet Syndrome News

Bringing together patients and their families — virtually, this year — the Dravet Syndrome Foundation (DSF) will present its fourth annual Day of Dravet workshop Oct. 17. The free event ... Read more

September 29, 2020September 29, 2020

Fycompa May Open to Younger Children With Select Seizures in Europe

Dravet Syndrome News

The Committee for Medicinal Products for Human Use (CHMP), an arm of the European Medicines Agency (EMA), recommended lowering the age limit of the anti-seizure medication Fycompa (perampanel) to 4 for children with focal-onset seizures and to ... Read more

September 22, 2020September 22, 2020

Fintepla Reduces Seizure Frequency in Dravet Syndrome Youngsters, Pivotal Phase 3 Trial Reports

Dravet Syndrome News

Fintepla (fenfluramine) oral solution, developed by Zogenix, significantly reduced seizure frequency in children and teenagers with Dravet syndrome, according to top-line results from a Phase 3 clinical trial. This trial ... Read more

September 15, 2020September 15, 2020

Fintepla Unlikely to Cause Heart Valve Problems, PAH in Young Patients, Extension Study Finds

Dravet Syndrome News

Children with Dravet syndrome receiving Fintepla (fenfluramine) to control their seizures are unlikely to develop cardiac valvulopathy — a disease in which at least one of the four heart valves ... Read more

September 8, 2020September 8, 2020

Soticlestat Significantly Reduces Seizure Frequency in Children, Top-line Data Show

Dravet Syndrome News

The investigational oral therapy Soticlestat (OV935/TAK-935), developed by Ovid Therapeutics and Takeda Pharmaceuticals, safely and effectively lowers seizure frequency in children with Dravet syndrome and Lennox‑Gastaut syndrome (LGS), according to top-line ... Read more

August 25, 2020August 26, 2020

First Patient Dosed in MONARCH Clinical Trial Testing Stoke’s STK-001

Dravet Syndrome News

The first patient has been dosed in Stoke Therapeutics‘ Phase 1/2a MONARCH trial, which is investigating the safety, tolerability, and efficacy of STK-001 as a treatment for children and adolescents with ... Read more

August 24, 2020September 24, 2020

Xperiome Platform Aims to Streamline Searches for Rare Diseases, Gain Patient Input

Dravet Syndrome News

Raremark, an online rare disease patient community, has launched a digital platform called Xperiome, aimed at streamlining the search for new medicines for rare disorders and incorporating more patient input ... Read more

August 18, 2020August 18, 2020

Epilepsy Foundation’s Community Day is Virtual This Year

Dravet Syndrome News

The Epilepsy Foundation’s Community Day, being held virtually this year, will offer patients, caregivers, and advocates the latest information about epilepsy and an opportunity to connect with others. “Innovation and Action ... Read more

August 17, 2020September 18, 2020

EveryLife Foundation Launches Scholarship Fund for Rare Disease Community in US

Dravet Syndrome News

The EveryLife Foundation for Rare Diseases has launched a scholarship fund in the U.S. to support individuals with rare disorders who are pursuing personal goals through training and education. The ... Read more

August 11, 2020August 11, 2020

SCN1A Gene Activation Reduced Seizures, Improved Behavior in Dravet Mice

Dravet Syndrome News

Activating the SCN1A gene, which is deficient in most patients with Dravet syndrome, reduced febrile seizures and improved behaviors in a mouse model, according to a recent study. The findings ... Read more

August 10, 2020September 10, 2020

New Streaming Channel Showcases Rare Disease Films

Dravet Syndrome News

When the COVID-19 pandemic forced the postponement of a rare disease film festival originally slated for May, its organizers set out to find a new way to bring the films ... Read more

August 4, 2020August 4, 2020

Epidiolex Now Available in U.S. for Dravet, LGS Patients Age 1 and Older

Dravet Syndrome News

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