News

The Australian Therapeutic Goods Administration (TGA) has approved Epidyolex (cannabidiol) as a treatment for seizures associated with Lennox-Gastaut syndrome (LGS) or Dravet syndrome in patients 2 or older, according to developers GW Pharmaceuticals. In order to make Epidyolex available to Australian patients,…

Same But Different, a nonprofit U.K. group that uses art for social change, is inviting people to choose their favorite photographs in a calendar contest to heighten awareness of rare diseases, including amyotrophic lateral sclerosis (ALS). The organization’s panel of judges has pared the number of contest submissions…

Eton Pharmaceuticals has submitted a new drug application (NDA) for topiramate oral solution — an anticonvulsant formerly known as ET-101 — to the U.S. Food and Drug Administration (FDA). The NDA covers three indications: as an add-on therapy for treatment of partial-onset seizures, including…

The Scottish Medicines Council (SMC) approved Epidyolex (cannabidiol), combined with the anti-epileptic medicine clobazam, as a treatment option for children and adults with Lennox-Gastaut or Dravet Syndrome.

A European Medicines Agency (EMA) committee has recommended that the age range of Fycompa (perampanel) be expanded in the European Union (EU) to include children 4 and older with focal-onset seizures and those 7 and older with primary generalized tonic-clonic seizures (PGTCS). In the EU, Fycompa — available in…

When used alongside valproic acid, Epidiolex (cannabidiol) can increase the risk of thrombocytopenia in children and adolescents with drug-resistant forms of epilepsy, such as Lennox-Gastaut syndrome (LGS), a study shows. According to the study authors, young patients with these disorders and receiving this combination of anti-seizure medications…

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 —…