educational resources
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A small, curated library of things worth reading, watching, or saving. We’ll keep this useful rather than exhaustive.
STORIES OF PROGRESS
Reasons for hope
Stories from beyond PKAN about rare-disease research, families, patients, and scientists pushing treatments forward. A reminder that diseases once thought untreatable can move in new directions.
keep this one ↘
01
HUFFPOST · ARTICLE
Cockayne syndrome
Gene therapy,
How David Fajgenbaum
Cockayne syndrome
+ gene therapy
Efforts to develop gene therapy for a rare genetic disorder.
read ↗ 02 THE NEW YORK TIMES · JULY 2026Gene therapy,
rare diseases
+ epilepsy
Emerging approaches for rare diseases, including severe forms of epilepsy.
read ↗ 03 RETHINKING · PODCASTHow David Fajgenbaum
saved his own life
A rare-disease experience became a mission to find overlooked treatments.
listen ↗PAPERS + ARTICLES
PKAN library
We are building a collection of useful PKAN papers, articles, PDFs, guides, and explainers. Scientific resources will include a short plain-language note about what they cover and why they may be useful.
last reviewed · august 2026