News

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…

Cannabidiol (CBD) — the active ingredient in Epidiolex — was found to bind to and block the opening of the sodium channel protein associated with seizures in people with Lennox-Gastaut syndrome (LGS), a study has shown. The findings describe the…

The two COVID-19 vaccines that recently received emergency approval from the U.S. and other worldwide regulatory agencies are expected to pose little risk to the rare disease community, including to patients with compromised immune systems or those participating in gene therapy studies. That was the message of a recent…

Caring for a loved one with a rare disease, especially during these uncertain times, demands significant time, attention, patience, and dedication. To help meet that need, the National Organization for Rare Disorders (NORD)’s Rare Caregiver Respite Program may be a helpful resource. The program seeks to give a…

Telehealth visits — conferencing with patients via video or phone calls — are generally viewed favorably by both the treating physicians and the parents of children with epileptic conditions like Lennox-Gastaut syndrome (LGS), a French study reports. The study, “Usefulness, limitations, and parental opinion about teleconsultation…

Having a sibling with Lennox-Gastaut syndrome and other seizure-causing conditions can take a psychological toll that is not always recognized by parents, research shows. The findings were published in Epilepsy and Behavior, in the study “Psychosocial impact on siblings of patients with developmental and epileptic encephalopathies.”…