STK-001, Stoke Therapeutics’ investigational therapy, continues to show promise in safely reducing seizures among children and adolescents with…
Articles by Patricia Inácio, PhD
Children and adolescents with Dravet syndrome with walking difficulties commonly experience impairments in functional mobility and greater restrictions in…
Note: This story was updated Feb. 7, 2022, to clarify that CAMP4 will continue preclinical studies for its lead candidate.
Single and multiple doses of Stoke Therapeutics’ investigational therapy STK-001 were well-tolerated and led to a significant reduction in…
The U.S. Food and Drug Administration (FDA) has granted orphan drug designation to Neuroene Therapeutics‘ NT102, a potential anti-seizure therapy for…
CAMP4 Therapeutics has entered an exclusive worldwide licensing agreement to develop, manufacture, and commercialize OPKO Health’s RNA-based technology…
The Dravet Syndrome Foundation (DSF) is accepting applications for its Patient Assistance Grants (PAG) program that provides financial…
Ahead of this year’s Rare Disease Week on Capitol Hill, held virtually July 14–22, the EveryLife Foundation will…
More than half of the people with rare diseases and their caregivers, asked in a survey, were undecided or less…
The EveryLife Foundation for Rare Diseases is accepting applications for a scholarship program that aims to help adults with a…