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Striking a Balanced View of Clinical Trials

This month marks 13 years since my sister’s diagnosis of CLN1 disease, or Batten disease. That means I’ve had 4,745 days — or 113,880 hours, or 6,832,800 minutes — to get used to the thought of outliving her, even though she came into the world on a warm August…

3.1 Ways to Fight Your Rare Disease

My life changed forever 13 years ago. In the space of a few months, I started a new job, bought my first house, got married, and searched “neuronal ceroid lipofuscinosis” (Batten disease) for the first time. I’ll never forget that phone call: I was writing a press release at…

My Road to Big Victories for CLN1 Disease

With 350 million patients, rare diseases represent the largest disease community on the planet. In fact, if all rare disease patients lived in the same country, they would represent the world’s third-largest country, behind only China and India. Of course, with so many included conditions (more than 7,000 and counting),…