Launch of SCN8A Clinicians’ Network

The first gathering of clinicians treating SCN8A children—which included leading neurologists from across the U.S. and Canada— concluded with a near unanimous commitment to collaborate on development of a Standard of Care for treating SCN8A.

The session was organized by FAST (Families Advancing SCN8A Treatments), a collaborative effort of two family foundations dedicated advancing scientific efforts to improving the treatment and outcomes for SCN8A children—the Shay Emma Hammer Research Foundation and Wishes for Elliott. Dr. John Schreiber, who runs an SCN8A clinic at Children’s National, joined Michael Hammer (SEHRF), Gabi Conecker and JayEtta Hecker (both with Wishes) to plan and host the gathering.

The discussion was kicked off with a detailed briefing providing “Perspectives of SCN8A Families on Clinical Care by Neurologists" (see the slides here). Family input was gathered from a structured survey instrument as well as unstructured interviews with multiple families – both conducted through the FAST network.

Detailed presentations were provided by:

- Dr. Hammer on insights from the dedicated SCN8A registry

- Dr. Schreiber on findings from the 22 children seen at the Children's National Medical Center Open SCN8A Clinic

- Dr. Ethan Goldberg on data curated from the 20+ patients seen at Children's Hospital of Philadelphia

Other important insights about process were provided by Dr. Elaine Wirrell, from Mayo Clinic, on her leadership of a similar clinical team that collaborated on the development of a Standard of Care for Dravet patients in 2016.

Other participants, bringing extensive experience and support for collaborating on a consensus document, included Dr. Ian Miller from Nicklaus Children's and Dr. Orrin Devinsky from NYU Langone, each with experience treating multiple SCN8A patients. Participants were enthusiastic about collaborative efforts to analyze the comprehensive data in the SCN8A registry, which many saw as an invaluable resource for moving forward. Clinicians began to explore efforts to share and integrate data on SCN8A patients across clinics.

Next steps will include a second meeting with additional clinicians treating SCN8A children.

Many thanks to all SCN8A families who demonstrated their commitment to advancing better treatments for our children through their participation in the FAST survey, network meetings and supporting a wide range of efforts to collaborate towards improved outcomes for our children.