News

Starting a 501(c)(3) tax-exempt nonprofit isn’t easy, but the National Organization for Rare Disorders gave a few tips for those  looking to begin the complex process in its Feb. 20 webinar.  William Whitman…

Fintepla (fenfluramine) reduced the frequency of convulsive seizures in children and adolescents with Dravet syndrome who responded poorly to treatment regimens that included Diacomit (stiripentol), Phase 3 trial results show. The findings were reported in a study, “Fenfluramine for Treatment-Resistant Seizures in…

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 the hundreds of millions of individuals they are thought to affect. Patients, caregivers, and advocates worldwide will sport denim ribbons…

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 a merged analysis of two Phase 3 trials. When given daily in addition to existing antiepileptic…

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 well tolerated in healthy volunteers. EPX-100 is a repurposed antihistamine, originally known as clemizole, which was…

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 resources, the FDA these days relies more and more on patients to…

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 two years after the European Union decided to relocate the EMA to the Netherlands in the wake of Brexit.

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 or acute repetitive seizures are challenging to treat and highly disruptive in the…

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 the Seizure program. New partners in the effort include Biogen, Encoded Therapeutics, Neurogene, Praxis…

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 these studies were presented by the company in a poster, titled “Targeted Augmentation of Nuclear Gene Output…