News

In recognition of Rare Disease Day Feb. 29, Bionews Services launched a social media campaign last month asking patients to describe what makes them rare. Running Feb. 7–29, the #WhatMakesMeRare campaign was aimed at uplifting people with rare diseases by encouraging them to share their stories and perspectives. The…

A new small molecule was seen to stimulate certain brain receptors, called NMDA receptors, and lessen abnormal brain epileptic activity in mouse models of Dravet syndrome and Alzheimer’s disease. The treatment also helped mice recover part of their learning and memory abilities, the research team that developed the…

Beginning on Feb. 29, Rare Disease Day, chapters from notable scientific books and clinical review articles covering rare disorders will be available free-of-charge from Elsevier. The offer runs through April 30, and aims to supports work by researchers and clinicians into a better understanding of and treatments for rare diseases, as well…

The U.S. Food and Drug Administration (FDA) extended by three months the review period for Zogenix’s New Drug Application (NDA) for Fintepla (ZX008), an investigational anti-seizure therapy for patients with Dravet syndrome. The agency now expects to provide a decision by June 25 on whether…

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…