February 21, 2020February 21, 2020
Patients, Supporters Worldwide Recognizing Rare Disease Day 2020
News
An abundance of events are afoot around the world to mark Rare Disease Day 2020 on Feb. 29. The ... Read more
February 18, 2020February 18, 2020
Fintepla Daily Significantly Lowers Seizures in Young Dravet Patients, Trial Data Show
News
Daily treatment with Fintepla (a low-dose oral solution of fenfluramine), an investigational therapy by Zogenix, significantly reduces seizure frequency in ... Read more
February 11, 2020February 11, 2020
Epygenix Advancing EPX-100 to Phase 2 Trial in Dravet Patients, Based on Positive Phase 1 Results
News
Epygenix Therapeutics is planning to launch a Phase 2 clinical trial testing EPX-100 in patients with Dravet syndrome, after ... Read more
February 10, 2020February 10, 2020
FDA Efforts to Bring Patients into Treatment Decisions Focus of NORD Webinar
News
The U.S. Food and Drug Administration (FDA), a vast government bureaucracy, employs about 17,500 people and had a budget ... Read more
February 5, 2020February 5, 2020
With Post-Brexit Move to Amsterdam Complete, EMA Renews Rare Disease Focus
News
The gleaming new Dutch headquarters of the European Medicines Agency (EMA), fronting Domenico Scarlattilaan in Amsterdam’s suburban Zuidas business ... Read more
January 28, 2020January 28, 2020
FDA Approves Valtoco Nasal Spray to Treat Seizure Clusters in Epilepsy Patients, 6 and Older
News
The United States Food and Drug Administration (FDA) has approved Valtoco (diazepam nasal spray) as a rescue treatment for acute, repetitive seizures (seizure ... Read more
January 21, 2020January 21, 2020
Behind the Seizure Program Expands Access to Free Genetic Testing for Children Under 8
News
To speed diagnoses and provide free genetic testing for young children in the United States and Canada who have ... Read more
January 14, 2020January 14, 2020
Stoke’s STK-001 Prolongs Survival, Reduces Seizures in Dravet Mouse Model, Preclinical Study Shows
News
STK-001, Stoke Therapeutics’s investigational therapy for Dravet syndrome, prolongs survival and reduces the frequency of seizures in a mouse ... Read more
January 7, 2020January 7, 2020
U.K. Physicians Now Can Prescribe Epidyolex Cannabidiol Solution to Treat Dravet, LGS Patients
News
Since Jan. 6, physicians in England are able to prescribe the oral cannabidiol solution Epidyolex, in combination with clobazam, to ... Read more
December 17, 2019December 17, 2019
Febrile Seizures After Vaccination May Indicate Dravet, Study Shows
News
Infants who develop febrile seizures a short time after being vaccinated and are positive for a disease-causing mutation in ... 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 ... 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

February 21, 2020February 21, 2020

Patients, Supporters Worldwide Recognizing Rare Disease Day 2020

News

An abundance of events are afoot around the world to mark Rare Disease Day 2020 on Feb. 29. The activities are focused on heightening awareness about rare diseases and ... Read more

February 18, 2020February 18, 2020

Fintepla Daily Significantly Lowers Seizures in Young Dravet Patients, Trial Data Show

News

Daily treatment with Fintepla (a low-dose oral solution of fenfluramine), an investigational therapy by Zogenix, significantly reduces seizure frequency in children and teenagers with Dravet syndrome while demonstrating safety, according to ... Read more

February 11, 2020February 11, 2020

Epygenix Advancing EPX-100 to Phase 2 Trial in Dravet Patients, Based on Positive Phase 1 Results

News

Epygenix Therapeutics is planning to launch a Phase 2 clinical trial testing EPX-100 in patients with Dravet syndrome, after Phase 1 results showed the investigational therapy was safe and ... Read more

February 10, 2020February 10, 2020

FDA Efforts to Bring Patients into Treatment Decisions Focus of NORD Webinar

News

The U.S. Food and Drug Administration (FDA), a vast government bureaucracy, employs about 17,500 people and had a budget of $5.7 billion in 2019. Yet even with its enormous ... Read more

February 5, 2020February 5, 2020

With Post-Brexit Move to Amsterdam Complete, EMA Renews Rare Disease Focus

News

The gleaming new Dutch headquarters of the European Medicines Agency (EMA), fronting Domenico Scarlattilaan in Amsterdam’s suburban Zuidas business district, finally opened for business last month — just over ... Read more

January 28, 2020January 28, 2020

FDA Approves Valtoco Nasal Spray to Treat Seizure Clusters in Epilepsy Patients, 6 and Older

News

The United States Food and Drug Administration (FDA) has approved Valtoco (diazepam nasal spray) as a rescue treatment for acute, repetitive seizures (seizure clusters) in people with epilepsy, ages 6 and older. “Cluster ... Read more

January 21, 2020January 21, 2020

Behind the Seizure Program Expands Access to Free Genetic Testing for Children Under 8

News

To speed diagnoses and provide free genetic testing for young children in the United States and Canada who have unexplained seizures, BioMarin Pharmaceutical and Invitae have expanded their Behind ... Read more

January 14, 2020January 14, 2020

Stoke’s STK-001 Prolongs Survival, Reduces Seizures in Dravet Mouse Model, Preclinical Study Shows

News

STK-001, Stoke Therapeutics’s investigational therapy for Dravet syndrome, prolongs survival and reduces the frequency of seizures in a mouse model of the disease, according to preclinical results. Findings from ... Read more

January 7, 2020January 7, 2020

U.K. Physicians Now Can Prescribe Epidyolex Cannabidiol Solution to Treat Dravet, LGS Patients

News

Since Jan. 6, physicians in England are able to prescribe the oral cannabidiol solution Epidyolex, in combination with clobazam, to treat persistent seizures in eligible people with Dravet syndrome and Lennox ... Read more

December 17, 2019December 17, 2019

Febrile Seizures After Vaccination May Indicate Dravet, Study Shows

News

Infants who develop febrile seizures a short time after being vaccinated and are positive for a disease-causing mutation in the SCN1A gene should be considered for a diagnosis of ... Read more