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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…
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 Gastaut…
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 Dravet syndrome, a new study shows. The study, “SCN1A variants in vaccine-related febrile seizures: a prospective study,” was published in…
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 Summit. From diagnosis and clinical trial design to manufacturing, pricing strategies, and ethical concerns, gene therapy — both its high…
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