Patient leadership and partnerships accelerate therapies for SCN8A and other developmental and epileptic encephalopathies.

• Tác giả: Gabrielle Conecker, Michael F Hammer, JayEtta Hecker
• Ngôn ngữ: eng
• Thông tin xuất bản: England : Therapeutic advances in rare disease , 2025
• Bộ sưu tập: NCBI
• ID: 664421

Tóm tắt

Families are a driving force in accelerating the understanding and science of SCN8A. The urgency felt by families facing the absence of treatments for their children makes them uniquely positioned to advance therapies through advocacy, data sharing, and partnerships. The International SCN8A Alliance (Alliance) brings families together to collaborate on advancing the science of SCN8A. The Alliance hosts SCN8A