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ET-101, Eton Pharmaceuticals’ proprietary topiramate oral solution shows similar properties and is biologically equivalent to the currently approved topiramate oral solid product, according to data from a bioequivalence study conducted in healthy volunteers. ET-101 is being investigated as a potential add-on therapy to manage seizures in…

The U.S. Drug Enforcement Administration (DEA) has reclassified Epidiolex, an oral cannabidiol solution used to treat seizures, as a non-controlled substance, potentially easing patient access to it. This decision removes all federal controlled-substance restrictions for Epidiolex, which was approved in 2018 by the U.S. Food and Drug Administration (FDA)…

Oral treatment with pharmaceutical cannabidiol (CBD), sold under the brand Epidiolex, seems to be more effective to control seizures than artisanal preparations of CBD, in children and young patients with Lennox-Gastaut syndrome (LGS) and other epilepsies, a preliminary study has found. The difference in seizure control “is…

First, the bad news: If you’re one of the 30 million or so Americans with a rare disease, you probably have lower immunity to the novel coronavirus than most people. Now, the good news: You already know how to face loneliness and adversity — qualities that make you far stronger…

Spontaneous mutations affecting a critical protein produced in nerve and muscle cells, called EEF1A2, were found to be the underlying cause of Lennox-Gastaut syndrome (LGS) in two boys in China, a study reports.  Its authors recommended EEF1A2 mutations be evaluated in LGS…

The Living Rare, Living Stronger Patient and Family Forum, originally set for May 14–16 in Cleveland, Ohio, has been postponed until July 18–20 because of the coronavirus disease COVID-19 pandemic. The event’s sponsor, the National Organization for Rare Disorders (NORD),…

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

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…

Banzel (rufinamide) — an approved add-on, anti-seizure oral therapy — is safe and effective in lowering seizure rates in children and adolescents with Lennox-Gastaut syndrome (LGS), according to a review study. Nevertheless, researchers point out that future studies are needed to better assess the therapy’s effectiveness and provide…