News

A new mouse model of Lennox-Gastaut syndrome (LGS) accurately reproduced key features of the disease and responded to approved and investigational therapies, providing the first pharmacological validation of the model, a new study reports. “These promising results suggest the mouse model could be a valuable tool for drug discovery…

A girl with difficult-to-treat CDKL5 deficiency disorder (CDD), whose epilepsy later developed into Lennox–Gastaut syndrome (LGS), became seizure-free for more than 11 months after treatment with low-dose Fintepla (fenfluramine), suggesting this medication may be a potential option for similarly hard-to-treat seizures, according to a report from Japan. After starting…

The Lennox-Gastaut Syndrome (LGS) Foundation has launched a collaborative research accelerator to unite experts, dismantle research roadblocks, and speed up the development of new treatments for LGS and related developmental and epileptic encephalopathies (DEEs). The newly formed LGS and Associated DEE Research Accelerator brings together pharmaceutical companies, academic…

A team of experts in Italy has proposed a framework for the routine monitoring of nonseizure symptoms in people with Lennox-Gastaut syndrome (LGS) throughout their lives. While the framework is a good place to start, the researchers noted a need for better assessment tools to adequately capture these symptoms and…

Children with Lennox-Gastaut syndrome (LGS) show significantly higher blood levels of proteins linked to nerve injury and cell death than healthy children, a small study in India suggests. Researchers discovered that these biomarker changes directly track with a heavier seizure burden, raising the possibility that simple blood tests could one…

People with rare disorders have a worse healthcare experience than those affected by chronic diseases, according to the results of an international survey conducted by Eurordis-Rare Diseases Europe. Indeed, rare disease patients overall give their healthcare experience a medium-low rating, of 2.5 on a scale of 1 to 5,…

Scores of virtual events are afoot around the world to mark Rare Disease Day 2021 on Feb. 28. 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…

International Epilepsy Day (IED), falling on Feb. 8 this year, calls attention to the needs of the roughly 50 million people globally who are affected by the brain disorder. “This is a day for everyone, no matter where you are, no matter how small your group or large your area,…

While progress was made last year on newborn screening and other policy issues critical to rare disease patients, a “State Report Card” argues that many concerns — notably out-of-pocket costs for prescription medicines and access to affordable comprehensive care — still need attention. Those were the findings of the…

Jazz Pharmaceuticals announced the signing of a definitive agreement for it to acquire GW Pharmaceuticals and holdings that include Epidiolex, an approved oral cannabinoid treatment for seizures in Lennox-Gastaut syndrome (LGS) patients. “We are excited to add GW’s industry-leading cannabinoid platform, innovative pipeline and products, which will strengthen and broaden…