December 10, 2019
Gene Therapy Takes Center Stage at 2019 NORD Summit
News
With so much recent publicity surrounding gene therapy, it’s no surprise that the topic was a major focus of ... Read more
December 5, 2019
HHS Secretary Alex Azar Touts White House Efforts to Cure Rare Diseases
News
Despite skyrocketing healthcare costs, President Trump is committed to protecting the 30 million or so Americans with rare diseases ... Read more
December 3, 2019December 3, 2019
StrideBio, Sarepta Partner to Advance Gene Therapies for Dravet, Other Rare Diseases
News
StrideBio and Sarepta Therapeutics announced a collaboration and license agreement to advance gene therapies for rare diseases of the central ... Read more
November 26, 2019November 26, 2019
Fintepla Lowers Seizure Frequency in Children and Teens with Dravet, Phase 3 Studies Report
News
Treatment with Fintepla (ZX008) can provide a clinically meaningful and profound reduction in the frequency of seizures in Dravet ... Read more
November 19, 2019November 19, 2019
NICE Favors Adding Epidyolex, Cannabidiol Oral Solution, to NHS for Dravet Patients in England
News
The National Institute of Health and Care Excellence (NICE), an arm of U.K.’s public health system, favors use of ... Read more
November 15, 2019November 16, 2019
Rare Disease Film Festival Highlights Patient and Researcher Unity
News
Rare disease-themed videos glowed on a large screen before an audience of people in wheelchairs, with crutches, and bearing ... Read more
November 12, 2019November 12, 2019
Fintepla Safely and Effectively Eases Seizures in Young Children with Dravet, Extension Study Finds
News
Treatment with Zogenix‘s Fintepla (ZX008) provides a sustained and meaningful reduction in convulsive seizure frequency in young children with ... Read more
November 6, 2019November 6, 2019
Treating Dravet Means Being Open to Variety of Care and Therapies, Review Study Says
News
Managing Dravet syndrome requires a multidisciplinary approach open to both pharmacological and non-pharmacological treatments, because of the severe nature of ... Read more
October 29, 2019October 29, 2019
NeuCyte, Trillium Therapeutics Team Up to Develop New Therapy for Drug-resistant Epilepsies
News
Neucyte and Trillium Therapeutics have established a license agreement to develop a new therapy for drug-resistant epilepsies, including Dravet ... Read more
October 22, 2019October 22, 2019
Soticlestat Can Reduce Seizure Frequency in Adults With Dravet, Other Rare Epilepsies, Initial Data Shows
News
Ovid Therapeutics‘ investigational treatment soticlestat (OV935/5/TAK935) was found to progressively reduce seizure frequency — by up to 90% — ... Read more
October 15, 2019October 15, 2019
Seizures in Dravet Patients May Be Associated with Abnormal Serotonin System, Preclinical Study Suggests
News
Seizures in people with Dravet syndrome may be associated with abnormal serotonin levels and activity in the brain, according ... Read more
October 8, 2019October 8, 2019
Cannabis-based Zygel Helps Reduce Seizures in Childhood Epilepsy Syndromes, Trial Reports
News
Zygel (ZYN002) — a cannabis-based gel formulation developed by Zynerba Pharmaceuticals — can significantly reduce seizures by up to ... Read more

December 10, 2019

Gene Therapy Takes Center Stage at 2019 NORD Summit

News

With so much recent publicity surrounding gene therapy, it’s no surprise that the topic was a major focus of the recent 2019 NORD Rare Diseases & Orphan Products Breakthrough ... Read more

December 5, 2019

HHS Secretary Alex Azar Touts White House Efforts to Cure Rare Diseases

News

Despite skyrocketing healthcare costs, President Trump is committed to protecting the 30 million or so Americans with rare diseases and ensuring timely, affordable access to lifesaving treatments, the nation’s ... Read more

December 3, 2019December 3, 2019

StrideBio, Sarepta Partner to Advance Gene Therapies for Dravet, Other Rare Diseases

News

StrideBio and Sarepta Therapeutics announced a collaboration and license agreement to advance gene therapies for rare diseases of the central nervous and neuromuscular systems, including Dravet syndrome. Many genetic conditions are ... Read more

November 26, 2019November 26, 2019

Fintepla Lowers Seizure Frequency in Children and Teens with Dravet, Phase 3 Studies Report

News

Treatment with Fintepla (ZX008) can provide a clinically meaningful and profound reduction in the frequency of seizures in Dravet syndrome patients, according to data from two Phase 3 clinical ... Read more

November 19, 2019November 19, 2019

NICE Favors Adding Epidyolex, Cannabidiol Oral Solution, to NHS for Dravet Patients in England

News

The National Institute of Health and Care Excellence (NICE), an arm of U.K.’s public health system, favors use of the oral cannabidiol solution Epidyolex as an add-on to clobazam ... Read more

November 15, 2019November 16, 2019

Rare Disease Film Festival Highlights Patient and Researcher Unity

News

Rare disease-themed videos glowed on a large screen before an audience of people in wheelchairs, with crutches, and bearing oxygen tanks this Nov. 9 and 10 in San Francisco. ... Read more

November 12, 2019November 12, 2019

Fintepla Safely and Effectively Eases Seizures in Young Children with Dravet, Extension Study Finds

News

Treatment with Zogenix‘s Fintepla (ZX008) provides a sustained and meaningful reduction in convulsive seizure frequency in young children with Dravet syndrome, with similar clinical efficacy to that seen in ... Read more

November 6, 2019November 6, 2019

Treating Dravet Means Being Open to Variety of Care and Therapies, Review Study Says

News

Managing Dravet syndrome requires a multidisciplinary approach open to both pharmacological and non-pharmacological treatments, because of the severe nature of this disease and its poor prognosis, a review study reports. ... Read more

October 29, 2019October 29, 2019

NeuCyte, Trillium Therapeutics Team Up to Develop New Therapy for Drug-resistant Epilepsies

News

Neucyte and Trillium Therapeutics have established a license agreement to develop a new therapy for drug-resistant epilepsies, including Dravet syndrome and related disorders. The agent, whose name is still undisclosed, ... Read more

October 22, 2019October 22, 2019

Soticlestat Can Reduce Seizure Frequency in Adults With Dravet, Other Rare Epilepsies, Initial Data Shows

News

Ovid Therapeutics‘ investigational treatment soticlestat (OV935/5/TAK935) was found to progressively reduce seizure frequency — by up to 90% — in adults with rare and hard-to-treat epilepsies, including Dravet syndrome, ... Read more