ABOUT
A neutral collaborative initiated by OPHN1 Foundation.
The ROCK Access Collaborative is being developed to help rare-disease organizations evaluate shared RhoA–ROCK biology and, where justified, build common scientific, safety, and regulatory infrastructure.
Why OPHN1 Foundation started this effort
OPHN1 is a Rho GTPase-activating protein involved in regulation of RhoA signaling, actin dynamics, synaptic function, and neurodevelopment. The Foundation’s work on ROCK inhibition exposed challenges that are difficult for any ultra-rare organization to solve alone: drug supply, pediatric safety, biomarker development, regulatory strategy, funding, and access to specialized expertise.
The collaborative is intended to determine where those challenges—and their solutions—can responsibly be shared across disorders.
Principles
- Evidence before advocacy for a specific drug
- Transparent inclusion and prioritization criteria
- Scientific independence and conflict disclosure
- Patient and family priorities built into decision-making
- Disease-specific control of data and development decisions
- Responsible communications that distinguish hypothesis from proof