For Organizations

FOR PATIENT ORGANIZATIONS

Join without having to endorse a treatment before the evidence is ready.

Organizations can participate at different levels—from observing and sharing information to co-developing research, safety, and access infrastructure.

Observer

Receive updates, attend open briefings, and follow the evidence landscape without making a formal scientific or financial commitment.

Scientific member

Nominate a scientific representative, contribute disease-specific knowledge, and participate in readiness assessment and research planning.

Development partner

Help fund or execute defined preclinical, biomarker, regulatory, safety, or clinical-development projects.

What your organization brings

  • Family priorities and lived experience
  • Scientific and clinical relationships
  • Registries, natural-history efforts, or contact networks
  • Available disease models and unpublished knowledge
  • Fundraising, advocacy, and communications capacity