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One Month Into Testing the Waters: What We've Learned
Glafabra
A month ago, this was a handful of posts and a hope that people would care about a disease most of them had never heard of. Since then: real questions on this portal, real conversations, and people who registered interest before we'd even finished explaining why.
That's not nothing. Rare disease work runs on people willing to pay attention before there's a finished product to point to.
Thank you f...more
What Is Enzyme Replacement Therapy for Fabry Disease?
Every two weeks, for life. That's the rhythm of enzyme replacement therapy (ERT) for Fabry disease — an IV infusion, several hours, indefinitely.
It works. ERT has extended and improved life for thousands of Fabry patients since it was approved. But it's also a permanent fixture on the calendar, and it doesn't fully reach every organ the disease affects.
Live-cel is built around a different questi...more
What's New in Glafabra's Testing the Waters Campaign
One month into Testing the Waters. Here's what's changing for month two.
More real faces. Starting this Tuesday, we're introducing you to the people building Glafabra — one team member at a time, starting with our CEO.
DECODE keeps going, but simpler: no more separate answer-comment days later. Each new riddle now opens with last time's answer, right in the post.
And more of Glafabra's actual stor...more
How Is Fabry Disease Inherited? — DECODE No. 10
(Answer to the question last time on August 3: Pompe disease and Gaucher disease — the same Live-cel platform, one gene swapped in, reaches both.)
Fabry disease runs in families, but not the way most genetic conditions do. One chromosome carries the whole story — and it changes who's affected depending on whether it's inherited from mom or dad.
What inheritance pattern is this? Drop your guess in ...more
One Platform, Three Rare Diseases — DECODE No. 9
(Last time: five patients, five years, durable enzyme — the FACTS proof.)
Here's the twist we've been building toward. The approach behind Live-cel for Fabry isn't limited to one disease. Swap the corrected gene, keep everything else, and the same platform takes aim at two more inherited enzyme disorders — one that weakens muscle, one that affects the spleen, liver, and bone.
Can you...more
