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Why Do All Daughters of an Affected Father Inherit Fabry Disease? — DECODE No. 11
Glafabra
(Answer to the question last time on August 6: Fabry disease is X-linked — the GLA gene sits on the X chromosome, so the pattern plays out differently for men and women.)
Here's the quirk that surprises people. A father with Fabry disease passes the condition to every single one of his daughters — but to none of his sons.
Why does it break down exactly that way? Drop your guess in the comments bel...more
One Month Into Testing the Waters: What We've Learned
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
Five Patients, Five Years: The Fabry Gene Therapy Data — DECODE No. 8
(Last time: CD34+ hematopoietic stem cells, the engine of Live-cel.)
Here's the number that anchors everything. In the FACTS trial, this many patients were followed for five years — and every one still had therapeutic enzyme levels at the end, with zero product-attributable serious adverse events.
How many patients? Drop your guess in the comments below.
Follow the whole serie...more
The Treatment Routine Fabry Patients Call 'Two-Week Increments' — DECODE No. 4
(Last time: cross-correction — fix some cells, they help the rest.)
Patients describe it as "living in two-week increments." It isn't a figure of speech — it's a calendar that never ends. Every other week. For life.
What treatment routine are they describing? Drop your guess in the comments below.
Follow the whole series: https://bit.ly/Glafabra-TTW
@Brian Christie @Nev...more
The Blood Marker That Tracks Fabry Disease — DECODE No. 2
(Last time: the missing enzyme in Fabry is alpha-galactosidase A.)
When that enzyme goes missing, one particular molecule builds up in the blood and becomes the fingerprint doctors track to measure the disease. Cut it roughly in half, and you've shown a therapy is working.
What's this telltale biomarker? Drop your guess in the comments below.
Follow the whole series: https://b...more
