Patricia Inácio, PhD, science writer —

STK-001, Stoke Therapeutics’ investigational therapy, continues to show promise in safely reducing seizures among children and adolescents with Dravet syndrome, according to new interim data from the ongoing Phase 1/2a MONARCH and ADMIRAL trials. “We are encouraged by the data from our ongoing studies of STK-001, which continue…

Children and adolescents with Dravet syndrome with walking difficulties commonly experience impairments in functional mobility and greater restrictions in daily life activities, a study suggests. Its researchers recommend that physiotherapy be given patients to improve their gait, potentially allowing for better mobility in daily life. The study, “…

Note: This story was updated Feb. 7, 2022, to clarify that CAMP4 will continue preclinical studies for its lead candidate. Also, CAMP4 licensed the antisense oligonucleotides from OPKO Health, not the RNA Actuating Platform, which was developed in-house. CAMP4 Therapeutics‘ investigational RNA-based therapy — called CMP-SCN (CO-3527) — increased…

Single and multiple doses of Stoke Therapeutics’ investigational therapy STK-001 were well-tolerated and led to a significant reduction in the frequency of convulsive seizures among children and adolescents with Dravet syndrome in the first three months of follow-up, interim data from the ongoing Phase 1/2a MONARCH trial show.

The U.S. Food and Drug Administration (FDA) has granted orphan drug designation to Neuroene Therapeutics‘ NT102, a potential anti-seizure therapy for Dravet syndrome. NT102, given orally, has been shown to prevent seizures in several animal models, with “excellent protection” against such attacks in Dravet syndrome, according to Neuroene. Orphan drug…

CAMP4 Therapeutics has entered an exclusive worldwide licensing agreement to develop, manufacture, and commercialize OPKO Health’s RNA-based technology as potential disease-modifying treatments for Dravet syndrome. As part of the deal, CAMP4 has prioritized OPKO’s lead candidate for Dravet to enter clinical development. “We are delighted to enter into…

The Dravet Syndrome Foundation (DSF) is accepting applications for its Patient Assistance Grants (PAG) program that provides financial aid to people with Dravet syndrome who need medical equipment, therapy devices and educational tools. Since 2009, the PAG program runs from March 1 to Dec. 1 or until the funds…

Ahead of this year’s Rare Disease Week on Capitol Hill, held virtually July 14–22, the EveryLife Foundation will award grants to top advocates of rare disease organizations who participate in the week’s pre-events. The top 50 point-earners will be eligible to win $1,000 to $5,000in  grants, totaling up…

More than half of the people with rare diseases and their caregivers, asked in a survey, were undecided or less than willing to be vaccinated for COVID-19 if a vaccine was approved under emergency use authorization instead of the routine process, the EveryLife Foundation for Rare Disease reports. These findings…

The EveryLife Foundation for Rare Diseases is accepting applications for a scholarship program that aims to help adults with a rare disease pursue personal goals through training and education. For a second year, the #RAREis Scholarship Fund — supported by Horizon Therapeutics – will award 35 one-time scholarships, each…