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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.

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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.

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Data Sovereignty

Preventing AI medicine's biggest trap for rare disease

Why AI drug discovery becomes extraction without ownership infrastructure.

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The rare disease singularity
Vision

The rare disease singularity

Four capabilities converged. Cures are reachable.

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VisionRegulatoryData SovereigntyUltra-rareAccelerating innovationEhlers-DanlosNewborn screeningCompanyHistoryPatient ExperienceScience & Data
Read all109 posts
  1. Jun 1, 2026It is no longer good business to leave rare diseases behind
  2. May 27, 2026Our FDA Comment: AI-Enabled Optimization of Early-Phase Clinical Trials Pilot Program
  3. May 25, 2026Preventing AI medicine's biggest trap for rare disease
  4. May 14, 2026The rare disease singularity
  5. May 7, 2026Ethan Perlstein and the other n-of-1
  6. May 7, 2026Adrian Krainer and the splicing mechanism that became Spinraza
  7. May 7, 2026Wendy Chung and the natural-history-first model
  8. May 7, 2026Steven Gray and the AAV9 platform for rare brain disease
  9. May 7, 2026Stanley Crooke and the n-of-many foundation
  10. May 7, 2026Timothy Yu and the lab that built the first individualized drug
  11. May 7, 2026AAV gene therapy at single-patient scale
  12. May 7, 2026How splice-switching ASOs work
  13. May 7, 2026What an n-of-1 trial actually is
  14. May 7, 2026The speed thesis
  15. May 7, 2026The community review as clinical trial
  16. May 7, 2026The cure horizon
  17. May 7, 2026The acceleration equation
  18. May 7, 2026The rare disease data market
  19. May 7, 2026The provider discovery problem
  20. May 7, 2026The global natural history study
  21. May 7, 2026High consanguinity as research accelerator
  22. May 7, 2026The regulatory arbitrage opportunity
  23. May 7, 2026Rare disease as the proving ground
  24. May 7, 2026The EDS diagnosis problem solves itself
  25. May 7, 2026The real-time trial as default
  26. May 7, 2026The product review as outcomes data
  27. May 7, 2026The insurance argument that wins
  28. May 7, 2026The end of the diagnostic odyssey
  29. May 7, 2026The phenotype cloud
  30. May 7, 2026The incidental diagnosis cascade
  31. May 7, 2026Control data for the next cure
  32. May 7, 2026Cross-condition signal detection
  33. May 7, 2026From Orphan Drug to Platform Drug
  34. May 7, 2026How Data Cures Rare Disease
  35. May 7, 2026The Drug Made for One
  36. May 7, 2026What the Nurses' Health Study Built, and What Rare Disease Lacks
  37. May 7, 2026Sponsors Leave. Patients Don't.
  38. May 7, 2026The Economics of One
  39. May 7, 2026The N-of-1 Trial as Infrastructure
  40. May 7, 2026The Parent Scientist
  41. May 7, 2026The Time Value of Data
  42. May 7, 2026When Diagnostic Categories Dissolve
  43. May 7, 2026Why We Say Cure
  44. May 7, 2026Glutaric acidemia I and the striatum
  45. May 7, 2026The C5-OH cluster
  46. May 7, 2026Isovaleric acidemia, the genotype-phenotype question
  47. May 7, 2026The fatty acid oxidation family
  48. May 7, 2026Carnitine uptake defect
  49. May 7, 2026LCHAD deficiency
  50. May 7, 2026Trifunctional protein deficiency
  51. May 7, 2026VLCADD, the three phenotypes
  52. May 7, 2026Hearing loss, the other newborn screen
  53. May 7, 2026Propionic acidemia, the cardiomyopathy the screen cannot prevent
  54. May 7, 2026Methylmalonic acidemia, mutase deficiency
  55. May 7, 2026Methylmalonic acidemia, cobalamin disorders
  56. May 7, 2026Argininosuccinic aciduria
  57. May 7, 2026Citrullinemia type I
  58. May 7, 2026Hurler syndrome, the bone marrow transplant pioneer
  59. May 7, 2026Hunter syndrome, the blood-brain barrier problem
  60. May 7, 2026GAMT, the third OTC supplement treatment
  61. May 7, 2026Hemoglobin SC, the quieter sickle variant
  62. May 7, 2026Sickle beta-thalassemia, the third form
  63. May 7, 2026CCHD, the screen without a blood spot
  64. May 7, 2026Duchenne, the other disorder waiting for the panel
  65. May 7, 2026CAH, the endocrine emergency the screen prevents
  66. May 7, 2026Tyrosinemia type I, the drug that rewrote the prognosis
  67. May 7, 2026Homocystinuria, the vitamin-responsive amino acid disorder
  68. May 7, 2026Krabbe disease and the screen that runs out of time
  69. May 7, 2026X-ALD, from Lorenzo's oil to gene therapy
  70. May 7, 2026The 14 EDS subtypes, in plain words
  71. May 7, 2026Vascular EDS, the dangerous subtype
  72. May 7, 2026Classical EDS, the original
  73. May 7, 2026Kyphoscoliotic EDS
  74. May 7, 2026Cardiac-valvular EDS
  75. May 7, 2026Dermatosparaxis EDS
  76. May 7, 2026Biotinidase deficiency, the vitamin that prevents deafness
  77. May 7, 2026Cystic fibrosis, the poster child grows up
  78. May 7, 2026Metachromatic leukodystrophy, the four million dollar treatment that needs screening
  79. May 7, 2026The Beighton Score Problem
  80. May 7, 2026EDS and pregnancy
  81. May 7, 2026Lidocaine resistance and EDS
  82. May 7, 2026PKU in the developing world
  83. May 7, 2026The Diagnostic Odyssey as Systems Failure
  84. May 7, 2026Three Specialists Who Don't Talk to Each Other
  85. May 7, 2026The Screening Success Nobody Talks About
  86. May 7, 2026When Perfect Compliance Is Not Enough
  87. May 7, 2026The Babies Whose Deaths Were Blamed on Their Parents
  88. May 7, 2026The Mennonite Clinic
  89. May 7, 2026The Mothers Who Built Newborn Screening
  90. May 7, 2026Bubble Boy to Population Screening in One Generation
  91. May 7, 2026Race, Screening, and the $2 Million Cure
  92. May 7, 2026The Father Who Became a Biotech CEO
  93. May 7, 2026The Most Expensive Drug and the Argument for Speed
  94. Apr 28, 2026Serola SI belt, a daily-life spotlight
  95. Apr 28, 2026PKU formula, a logistics primer
  96. Apr 28, 2026What is newborn screening, in plain words
  97. Apr 28, 2026hEDS criteria 2017, for the newly diagnosed
  98. Apr 28, 2026The network, and why it exists
  99. Apr 22, 2026Our FDA Comment: Plausible Mechanism Framework Draft Guidance
  100. Apr 22, 2026Our Founder
  101. Apr 22, 2026The Data That Saves: How Robert Guthrie Built the Infrastructure for a Cure
  102. Apr 22, 2026What "Best Case" Looks Like in Rare Disease
  103. Apr 22, 2026From N-of-1 to Engine of Cures
  104. Apr 22, 2026Small Datasets Are Not Small When They Are Dense
  105. Apr 22, 2026You Are Not a Subject
  106. Apr 22, 2026The FDA Is Ready. The Infrastructure Is Not.
  107. Apr 22, 2026Cures, Not Management
  108. Apr 22, 2026We Are Not 23andMe
  109. Apr 22, 2026The Regulatory Landscape Is Finally Ready