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BioNews, the publisher of this website, hosted a virtual panel discussion on Rare Disease Day 2022, taking a deeper dive into what it’s like to live with a rare disease, including conversations about advocacy, mental health, survivor’s guilt, treatment of minority patients, and more. The Monday event, “A…
Treatment with soticlestat, an inhibitor of cholesterol turnover in the brain, protected against fever-induced seizures in two mouse models of Dravet syndrome, a study has found. In one of the models, the experimental therapy also prevented sudden unexpected death in epilepsy (SUDEP), a rare complication of seizures —…
A rare disease puts an economic burden on the patients, families, and caregivers that it affects, and will no doubt be an integral part of discussions on Rare Disease Day 2022, which brings international awareness about the more than 300 million people living with rare disorders. Part of that…
The nonprofit RARE-X is creating an easily-accessible, centralized data hub for all rare disease patient data that can help researchers answer questions about existing disorders, discover new ones, and work toward finding treatments. It was spun out of the work that Nicole Boice, founder and chief engagement officer of…
Eight adults with lifelong histories of epilepsy and developmental delays without any known cause were diagnosed with Dravet syndrome after undergoing genetic testing, a new study reports. The finding suggests “that a number of adult patients are currently undiagnosed and have unmet health needs,” according to researchers. “In our…
It’s been nearly a year since the EveryLife Foundation for Rare Diseases released its expansive report finding the total economic burden of rare disorders in the U.S. to be nearly $1 trillion.
Pharmaceutical giant UCB has agreed to buy Zogenix, the developer of Fintepla (fenfluramine), an oral add-on medicine that treats seizures associated with Dravet syndrome, a rare type of epilepsy. The purchase, which could cost UCB up to about $1.9 billion, is a step toward expanding the company’s…
Since 2008, Rare Disease Day — the last day of February — has brought together patients, caregivers, family members, friends, and advocates from around the world to raise awareness and improve equity for the more than 7,000 known rare diseases that affect more than 300 million people. In 2022, the…
On the second Monday of every February, International Epilepsy Day supporters all over the world bring attention to the challenges and needs of the epilepsy community. This year, the event falls on Feb. 14. Participants in more than 130 countries are poised to mark the day — from sharing…
The Epilepsy Foundation’s Walk to END EPILEPSY fundraiser series returns in person this spring, along with its 2.6-Million-Mile-Challenge. The “Walk” series opens in April with a goal of raising $1,750,000 to help improve the lives of the 3.4 million U.S. residents thought to live with epilepsy, including those…
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