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Epidiolex (cannabidiol), as an add on-therapy administered at either a low or high dose, leads to similar clinically relevant reductions in the frequency of convulsive seizures in children with treatment-resistant Dravet syndrome, Importantly, a lower Epidiolex dose shows a better safety and tolerability profile when compared to the higher dose.

Even with the coronavirus pandemic ravaging Europe and much of the world, patient advocate Lucia Monaco, PhD, of Italy remains confident that the Paris-based nonprofit she chairs will see the approval of 1,000 new rare disease therapies by 2027. That group, the International Rare Diseases Research Consortium (IRDiRC) —…

The ELEKTRA clinical trial assessing the investigational oral therapy soticlestat (OV935/TAK-935) in children with Dravet syndrome and Lennox-Gastaut syndrome (LGS) has completed patient enrollment, Ovid Therapeutics recently announced. “We completed enrollment significantly ahead of schedule in our placebo-controlled Phase 2 ELEKTRA…

An antisense oligonucleotide therapy that lowers the amount of Scn8a transcripts — RNA molecules used as a template for the production of proteins — delayed seizure onset and prolonged the lifespan of mice in a model of Dravet syndrome and SCN8A encephalopathy, a study found. The study, “…

The number of treatments for children with rare diseases has grown over the past decade, according to a new study. However, despite the increase, nearly 7,000 rare diseases are still lacking treatment. And federal incentives to boost treatment development for these rare diseases have primarily focused not on creating new…

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…