News

Treatment with soticlestat (OV935/TAK-935) safely led to a clinically meaningful reduction in the frequency of hard-to-treat seizures in a group of children with Dravet and Lennox-Gastaut (LGS) syndromes, according to top-line data from a Phase 2 trial. Soticlestat is an experimental oral therapy…

Same But Different, a U.K. nonprofit that uses the arts to bring communities together, is holding a calendar photography competition to raise awareness for rare diseases. Under the theme “A Glimmer of Hope,” the competition is a means to “visually express the hope that exists for people affected by…

Raremark, an online rare disease patient community, has launched a digital platform called Xperiome, aimed at streamlining the search for new medicines for rare disorders and incorporating more patient input into research. The goal is to help the pharmaceutical industry deliver innovative new therapies faster and smarter, the…

The EveryLife Foundation for Rare Diseases has launched a scholarship fund in the U.S. to support individuals with rare disorders who are pursuing personal goals through training and education. The initial phase of the five-year, $1-million #RAREis Scholarship Fund will include 32 scholarships — each totaling $5,000 —…

The U.S. Food and Drug Administration (FDA) has expanded the age range of patients to whom Epidiolex (cannabidiol) can be prescribed, to include children as young as age 1, according to its developer, GW Pharmaceuticals (GW). The medication had previously been made available to patients ages 2…

The Epilepsy Foundation’s Community Day, being held virtually this year, will offer patients, caregivers, and advocates the latest information about epilepsy and an opportunity to connect with others. “Innovation and Action to End Epilepsy,” a free national conference on Aug. 29, will focus on advocacy and a variety…

When the COVID-19 pandemic forced the postponement of a rare disease film festival originally slated for May, its organizers set out to find a new way to bring the films to an audience.  Co-founders Daniel DeFabio and Bo Bigelow, who are both fathers of children with…

Epidiolex, a cannabidiol add-on medication for treatment-resistant epilepsy, was safely given to children and teenagers for one year in a clinical trial that included youth with Lennox-Gastaut syndrome, a study reported.  Seizure control was improved with no adverse treatment…

Epidiolex (cannabidiol) and clobazam showed no signs of adverse drug-drug interactions in treating people with epilepsy, according to the results of a recent Phase 2 study. The study, “A Phase 2, Double‐Blind, Placebo‐Controlled Trial to Investigate Potential Drug‐Drug Interactions Between Cannabidiol and Clobazam,” was published…

Using the cannabidiol Epidiolex in combination with Diacomit (stiripentol) or Depacon (sodium valproate) — two anti-epileptic treatments — does not seem to raise major safety concerns among patients with seizures caused by conditions such as Lennox-Gastaut syndrome (LGS), a Phase 2 study…