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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RegulatoryOur FDA Comment: Plausible Mechanism Framework Draft Guidance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.
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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 all10 of 106 · 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