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The case for patient-controlled rare disease data across science, policy, and the lived experience.
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Vision
It is no longer good business to leave rare diseases behind
The math changed. The institutions that retool first capture leadership.
Read →This week
Regulatory
Our FDA Comment: AI-Enabled Optimization of Early-Phase Clinical Trials Pilot Program
Cureledger's May 27, 2026 comment to Docket FDA-2026-N-4390: rare disease as the pilot's most generalizable use case, within-person variability as the decision-quality threshold, privacy for cohorts under 100, and data custody that outlives the sponsor.
Read →Data Sovereignty
Preventing AI medicine's biggest trap for rare disease
Why AI drug discovery becomes extraction without ownership infrastructure.
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Vision
The rare disease singularity
Four capabilities converged. Cures are reachable.
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Clear ×Read all10 of 109 · Tag: Ultra-rare
- May 7, 2026Ethan Perlstein and the other n-of-1
- May 7, 2026Adrian Krainer and the splicing mechanism that became Spinraza
- May 7, 2026Wendy Chung and the natural-history-first model
- May 7, 2026Steven Gray and the AAV9 platform for rare brain disease
- May 7, 2026Stanley Crooke and the n-of-many foundation
- May 7, 2026Timothy Yu and the lab that built the first individualized drug
- May 7, 2026AAV gene therapy at single-patient scale
- May 7, 2026How splice-switching ASOs work
- May 7, 2026What an n-of-1 trial actually is
- Apr 28, 2026The network, and why it exists