News

The risk of severe COVID-19 outcomes does not appear to increase in people with Dravet syndrome, although they still may need additional medical attention, according to the results of a recent survey in the U.K. Results of the survey, “The impact of COVID‐19 in Dravet syndrome:…

A $200,000 Comcast NBCUniversal grant has been awarded to The Arc to increase access to educational resources for students of color and economically disadvantaged youngsters with intellectual and developmental disabilities (IDD), which can include Dravet syndrome. The award will fund the organization’s community-based The Arc@School initiative to expand its resources…

Jazz Pharmaceuticals is set to acquire GW Pharmaceuticals and gain access to a diversified pipeline of cannabis-based medications, including Epidiolex (cannabidiol), which is approved to treat seizures in patients with therapy-resistant forms of epilepsy, such as Dravet syndrome. The transaction, which involves a cash-and-stock deal totaling…

Diagnosed with sickle cell disease as a 6-month-old, Tristan Lee has faced a lot of challenges over his 37 years of life. But from a young age, he also learned how to turn those trials into triumphs. At age 9, a stroke due to his disease left him paralyzed…

Rare Disease Day at NIH, organized by the National Institutes of Health (NIH) and taking place on March 1, will feature panel discussions, patient stories, research updates, TED-style talks, and a presentation by a Nobel laureate recently recognized for her work on a gene editing tool. The free, virtual…

If a Maryland bill called Brynleigh’s Act becomes law, the state will join five others — all of which have passed “seizure safe schools” measures — in making sure every school is equipped with the tools necessary to provide a safe learning environment for students with epilepsy. The law…

People with rare disorders have a worse healthcare experience than those affected by chronic diseases, according to the results of an international survey conducted by Eurordis-Rare Diseases Europe. Indeed, rare disease patients overall give their healthcare experience a medium-low rating, of 2.5 on a scale of 1 to 5,…

Diacomit (stiripentol) is safe and effective at reducing the frequency of different types of seizures in people with Dravet syndrome when used routinely, including up to about two years, according to interim data from an ongoing study of its real-life use in Japan. Findings from the study, “…

Scores of virtual events are afoot around the world to mark Rare Disease Day 2021 on Feb. 28. The activities are focused on heightening awareness about rare diseases and the hundreds of millions of individuals they are thought to affect. Patients, caregivers, and advocates worldwide will sport denim ribbons…

After its approval in Europe in December, Fintepla (fenuramine) now is  commercially available in Germany as an add-on treatment for seizures associated with Dravet syndrome in patients 2 and older, the therapy’s developer, Zogenix, recently announced. “With the launch of FINTEPLA in Germany, physicians and caregivers here…