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The use of TANGO antisense oligonucleotide-based technology, developed by Stoke Therapeutics, resulted in a dose-dependent increase in brain levels of the NaV1.1 protein that is deficient in Dravet syndrome, a mouse study has shown. This technology can potentially treat the…
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…
Fintepla has no substantial impact on growth, in terms of height and weight, in children with Dravet syndrome, clinical trial data suggest. The findings were shared at the 2020 American Epilepsy Society (AES) Annual Meeting, in the presentation, “Treatment with Fintepla (fenluramine) in Patients with…
The National Organization for Rare Disorders (NORD) is seeking individuals willing to share real-life experiences with rare diseases to speak at its upcoming virtual Living Rare, Living Stronger NORD Patient and Family Forum. The interactive, patient-focused forum will be held online June 26-27. The deadline to apply for…
After some 59 years, the chief drug policy-making entity of the United Nations (UN) has voted to remove cannabis from a listing of narcotics designated as potentially addictive and dangerous, and having little to no therapeutic use. The reclassification by the Commission on Narcotic Drugs (CND) opens the door to…
Tevard Biosciences and Zogenix announced entering a collaboration agreement to advance potential gene therapies for genetic epilepsies, including Dravet syndrome. “We are pleased to announce our collaboration with Zogenix, whose commitment to developing new treatments for Dravet syndrome and other genetic epilepsies is unparalleled,” Daniel Fischer, co-founder,…
Zogenix’s Fintepla (fenfluramine) has been approved by the European Commission (EC) as an add-on treatment for seizures associated with Dravet syndrome in patients ages 2 and older. With this decision, Zogenix will be able to market Fintepla in all European Union (EU) member states, plus the United Kingdom,…
A no-cost genetic screening program, sponsored by Stoke Therapeutics, helped speed up molecular diagnosis of SCN1A-related diseases, including Dravet syndrome, by four years in children suspected of having genetic epilepsy, a study shows. Notably, clinical data from most of the children with SCN1A-related disorders suggested a future clinical diagnosis…
The U.S. Food and Drug Administration (FDA) will allow an additional higher dose of STK-001 to be added to the Phase 1/2a MONARCH trial, which is evaluating the potential therapy’s safety and tolerability in treating children and adolescents with Dravet syndrome. The FDA had previously placed higher…
To empower and equip members of the rare disease community to engage state leaders in matters of importance to patients and their families, the National Organization for Rare Disorders (NORD) has launched an initiative across the U.S. Its goal is to establish a Rare Disease Advisory Council (RDAC)…
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