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The case for patient-controlled rare disease data across science, policy, and the lived experience.
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RegulatoryOur FDA Comment: Plausible Mechanism Framework Draft Guidance
Cureledger’s April 22, 2026 comment to Docket FDA-2026-D-1256: natural history data standards, privacy for small populations, and data lifecycle, reuse, and custody under the PMF.
Read →This week
The Data That Saves: How Robert Guthrie Built the Infrastructure for a Cure
In 1960, a microbiologist solved two problems at once: detecting PKU and collecting the specimen from every newborn, everywhere, without requiring infrastructure most families would never access. We named our platform after him.
Read →Ultra-rareEthan Perlstein and the other n-of-1
The n-of-1 conversation is dominated by ASOs and AAV gene therapies that produce custom-built molecules. Perlstein's argument for the past decade is that small-molecule drug repurposing in model organisms can help many ultra-rare families faster and cheaper, when the biology cooperates.
Read →Newborn screeningWhat is newborn screening, in plain words
Heel-prick blood, a federal panel, and what state-by-state coverage actually looks like.
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Read all29 of 106 · Tag: Accelerating innovation
- May 7, 2026The speed thesis
- May 7, 2026The community review as clinical trial
- May 7, 2026The cure horizon
- May 7, 2026The acceleration equation
- May 7, 2026The rare disease data market
- May 7, 2026The provider discovery problem
- May 7, 2026The global natural history study
- May 7, 2026High consanguinity as research accelerator
- May 7, 2026The regulatory arbitrage opportunity
- May 7, 2026Rare disease as the proving ground
- May 7, 2026The real-time trial as default
- May 7, 2026The product review as outcomes data
- May 7, 2026The insurance argument that wins
- May 7, 2026The end of the diagnostic odyssey
- May 7, 2026The phenotype cloud
- May 7, 2026The incidental diagnosis cascade
- May 7, 2026Control data for the next cure
- May 7, 2026Cross-condition signal detection
- May 7, 2026From Orphan Drug to Platform Drug
- May 7, 2026How Data Cures Rare Disease
- May 7, 2026The Drug Made for One
- May 7, 2026What the Nurses' Health Study Built, and What Rare Disease Lacks
- May 7, 2026Sponsors Leave. Patients Don't.
- May 7, 2026The Economics of One
- May 7, 2026The N-of-1 Trial as Infrastructure
- May 7, 2026The Parent Scientist
- May 7, 2026The Time Value of Data
- May 7, 2026When Diagnostic Categories Dissolve
- May 7, 2026Why We Say Cure