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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…
Treatment for one year or longer with the antiseizure therapy Fintepla (fenfluramine) was shown to have a minimal impact on height and weight in children with Dravet syndrome, according to data from an open-label extension study. Despite Fintepla’s active ingredient being fenfluramine — a medicine initially…
Note: This story was updated Feb. 7, 2022, to clarify that CAMP4 will continue preclinical studies for its lead candidate. Also, CAMP4 licensed the antisense oligonucleotides from OPKO Health, not the RNA Actuating Platform, which was developed in-house. CAMP4 Therapeutics‘ investigational RNA-based therapy — called CMP-SCN (CO-3527) — increased…
Zogenix is seeking approval to market Fintepla (fenfluramine) in Japan as a treatment for epileptic seizures associated with Dravet syndrome. The company’s new drug application will be reviewed and decided on by the Japanese Ministry of Health, Labour, and Welfare. “There remains a substantial unmet need in…
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