More than 70% of Dravet syndrome (DS) patients demonstrated a reduction in seizure frequency of at least 50% while treated with Fintepla (fenfluramine) as an add-on therapy for up to one year, according to an analysis of real-world data. The antiseizure response to this medication was similar in…
The blood levels of Fintepla (fenfluramine) are mostly within the lower range among treated children and adults with Dravet syndrome and other epileptic conditions, a study has found. Still, these levels were enough to achieve good seizure control in most Dravet patients, further supporting the therapy’s potent effects,…
Treatment with soticlestat significantly reduced seizure frequency for children with Dravet syndrome or Lennox-Gastaut syndrome (LGS), another childhood disorder that causes seizures, in a Phase 2 clinical trial called ELEKTRA. Trial results were in the study, “A phase 2, randomized, double-blind, placebo-controlled study to evaluate the…
Up to three years of treatment with the anti-seizure medication Fintepla (fenfluramine) did not result in any signs of valvular heart disease — damage to any of the heart’s four valves — or pulmonary arterial hypertension (PAH) in children with Dravet syndrome. That’s according to new results from an…
Treatment with low-dose aripiprazole — a newer class of antipsychotic medication — in combination with psychotherapy, and behavioral and occupational therapy, was found to lessen behavior, attention, and educational problems in a 6-year-old girl with Dravet syndrome, according to a case study. This case points out a potentially effective…
Note: This story was updated July 13, 2022, to correct the name of Rare-X’s CEO Charlene Son Rigby. Nonprofits, scientists, governmental organizations, and the rare disease drug development industry have long cited 7,000 as the average number of rare diseases in the world.
Biocodex has chosen Pantherx Rare specialty pharmacy to be the sole U.S. distributor of Diacomit (stiripentol), an add-on and oral anti-seizure medicine that is approved for people with Dravet syndrome, ages 2 and older. The pharmacy, licensed in all 50 states, will add Diacomit to its portfolio…
The prevalence of Dravet syndrome in the U.K. is low, with most patients diagnosed before age 18, a large-scale healthcare database analysis showed. The findings also showed that the use of anti-seizure medications and healthcare resources was high among those with Dravet. This study provided valuable information on the…
The prevalence of Dravet syndrome in Sweden was one in 45,000 at the end of 2018, with most identified patients having a disease-causing mutation in the SCN1A gene, a study found. Data showed the rate of new diagnoses was higher, the age at diagnosis lower, and the use of contraindicated sodium…
An international panel of physicians and caregivers with expertise in Dravet syndrome is recommending genetic testing in children ages 2 to 15 months who develop normally but have a prolonged seizure after fever or vaccination. Among several consensus statements, the panel also recommended a number of currently available therapies…
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