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The case for patient-controlled rare disease data across science, policy, and the lived experience.

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Regulatory

Our FDA Comment: Plausible Mechanism Framework Draft Guidance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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History

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-rare

Ethan 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 screening

What 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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Cureledger mission

The case for patient-controlled rare disease data — science, policy, regulation.

12 posts

Founder blog

Nina Kilbride on building Cureledger and the company behind it.

1 posts

Zebra Reports

Community reviews of rare disease products, written by the people who use them.

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Read all106 posts
  1. May 7, 2026Ethan Perlstein and the other n-of-1
  2. May 7, 2026Adrian Krainer and the splicing mechanism that became Spinraza
  3. May 7, 2026Wendy Chung and the natural-history-first model
  4. May 7, 2026Steven Gray and the AAV9 platform for rare brain disease
  5. May 7, 2026Stanley Crooke and the n-of-many foundation
  6. May 7, 2026Timothy Yu and the lab that built the first individualized drug
  7. May 7, 2026AAV gene therapy at single-patient scale
  8. May 7, 2026How splice-switching ASOs work
  9. May 7, 2026What an n-of-1 trial actually is
  10. May 7, 2026The speed thesis
  11. May 7, 2026The community review as clinical trial
  12. May 7, 2026The cure horizon
  13. May 7, 2026The acceleration equation
  14. May 7, 2026The rare disease data market
  15. May 7, 2026The provider discovery problem
  16. May 7, 2026The global natural history study
  17. May 7, 2026High consanguinity as research accelerator
  18. May 7, 2026The regulatory arbitrage opportunity
  19. May 7, 2026Rare disease as the proving ground
  20. May 7, 2026The EDS diagnosis problem solves itself
  21. May 7, 2026The real-time trial as default
  22. May 7, 2026The product review as outcomes data
  23. May 7, 2026The insurance argument that wins
  24. May 7, 2026The end of the diagnostic odyssey
  25. May 7, 2026The phenotype cloud
  26. May 7, 2026The incidental diagnosis cascade
  27. May 7, 2026Control data for the next cure
  28. May 7, 2026Cross-condition signal detection
  29. May 7, 2026From Orphan Drug to Platform Drug
  30. May 7, 2026How Data Cures Rare Disease
  31. May 7, 2026The Drug Made for One
  32. May 7, 2026What the Nurses' Health Study Built, and What Rare Disease Lacks
  33. May 7, 2026Sponsors Leave. Patients Don't.
  34. May 7, 2026The Economics of One
  35. May 7, 2026The N-of-1 Trial as Infrastructure
  36. May 7, 2026The Parent Scientist
  37. May 7, 2026The Time Value of Data
  38. May 7, 2026When Diagnostic Categories Dissolve
  39. May 7, 2026Why We Say Cure
  40. May 7, 2026Glutaric acidemia I and the striatum
  41. May 7, 2026The C5-OH cluster
  42. May 7, 2026Isovaleric acidemia, the genotype-phenotype question
  43. May 7, 2026The fatty acid oxidation family
  44. May 7, 2026Carnitine uptake defect
  45. May 7, 2026LCHAD deficiency
  46. May 7, 2026Trifunctional protein deficiency
  47. May 7, 2026VLCADD, the three phenotypes
  48. May 7, 2026Hearing loss, the other newborn screen
  49. May 7, 2026Propionic acidemia, the cardiomyopathy the screen cannot prevent
  50. May 7, 2026Methylmalonic acidemia, mutase deficiency
  51. May 7, 2026Methylmalonic acidemia, cobalamin disorders
  52. May 7, 2026Argininosuccinic aciduria
  53. May 7, 2026Citrullinemia type I
  54. May 7, 2026Hurler syndrome, the bone marrow transplant pioneer
  55. May 7, 2026Hunter syndrome, the blood-brain barrier problem
  56. May 7, 2026GAMT, the third OTC supplement treatment
  57. May 7, 2026Hemoglobin SC, the quieter sickle variant
  58. May 7, 2026Sickle beta-thalassemia, the third form
  59. May 7, 2026CCHD, the screen without a blood spot
  60. May 7, 2026Duchenne, the other disorder waiting for the panel
  61. May 7, 2026CAH, the endocrine emergency the screen prevents
  62. May 7, 2026Tyrosinemia type I, the drug that rewrote the prognosis
  63. May 7, 2026Homocystinuria, the vitamin-responsive amino acid disorder
  64. May 7, 2026Krabbe disease and the screen that runs out of time
  65. May 7, 2026X-ALD, from Lorenzo's oil to gene therapy
  66. May 7, 2026The 14 EDS subtypes, in plain words
  67. May 7, 2026Vascular EDS, the dangerous subtype
  68. May 7, 2026Classical EDS, the original
  69. May 7, 2026Kyphoscoliotic EDS
  70. May 7, 2026Cardiac-valvular EDS
  71. May 7, 2026Dermatosparaxis EDS
  72. May 7, 2026Biotinidase deficiency, the vitamin that prevents deafness
  73. May 7, 2026Cystic fibrosis, the poster child grows up
  74. May 7, 2026Metachromatic leukodystrophy, the four million dollar treatment that needs screening
  75. May 7, 2026The Beighton Score Problem
  76. May 7, 2026EDS and pregnancy
  77. May 7, 2026Lidocaine resistance and EDS
  78. May 7, 2026PKU in the developing world
  79. May 7, 2026The Diagnostic Odyssey as Systems Failure
  80. May 7, 2026Three Specialists Who Don't Talk to Each Other
  81. May 7, 2026The Screening Success Nobody Talks About
  82. May 7, 2026When Perfect Compliance Is Not Enough
  83. May 7, 2026The Babies Whose Deaths Were Blamed on Their Parents
  84. May 7, 2026The Mennonite Clinic
  85. May 7, 2026The Mothers Who Built Newborn Screening
  86. May 7, 2026Bubble Boy to Population Screening in One Generation
  87. May 7, 2026Race, Screening, and the $2 Million Cure
  88. May 7, 2026The Father Who Became a Biotech CEO
  89. May 7, 2026The Most Expensive Drug and the Argument for Speed
  90. Apr 28, 2026Serola SI belt, a daily-life spotlight
  91. Apr 28, 2026PKU formula, a logistics primer
  92. Apr 28, 2026What is newborn screening, in plain words
  93. Apr 28, 2026hEDS criteria 2017, for the newly diagnosed
  94. Apr 28, 2026The network, and why it exists
  95. Apr 22, 2026Our FDA Comment: Plausible Mechanism Framework Draft Guidance
  96. Apr 22, 2026Our Founder
  97. Apr 22, 2026The Data That Saves: How Robert Guthrie Built the Infrastructure for a Cure
  98. Apr 22, 2026What "Best Case" Looks Like in Rare Disease
  99. Apr 22, 2026From N-of-1 to Engine of Cures
  100. Apr 22, 2026Small Datasets Are Not Small When They Are Dense
  101. Apr 22, 2026You Are Not a Subject
  102. Apr 22, 2026The FDA Is Ready. The Infrastructure Is Not.
  103. Apr 22, 2026Cures, Not Management
  104. Apr 22, 2026We Are Not 23andMe
  105. Apr 22, 2026The Regulatory Landscape Is Finally Ready
  106. Apr 22, 2026Henrietta Lacks at Scale