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Can Fabry Disease Occur With No Family History? — DECODE No. 12
Glafabra
Answer to the question last time on August 10: a father passes his only X chromosome to every daughter and his Y to every son — so every daughter inherits his Fabry variant, and no son does.)
Most Fabry cases run in families. But every so often, someone is diagnosed with zero family history at all — no parent, no sibling, nothing in the records.
What causes Fabry disease to show up with no family ...more
Why Does Live-cel Use Hematopoietic Stem Cells?
Why build a gene therapy around a blood stem cell instead of a virus alone?
A hematopoietic stem cell (HSC) — the cell that gives rise to your entire blood and immune system — can be corrected once, outside the body, and then put back in. It engrafts permanently, and its corrected descendants keep making the missing enzyme for years.
That's the foundation Live-cel is built on: harvest, correct, re...more
Meet Glafabra's CEO & Co-Founder, Chris Hopkins
Meet the person leading this: Chris Hopkins, CEO & Co-Founder.
Chris is a geneticist, biochemist, and entrepreneur who's spent his career at the intersection of science and company-building. He guided the acquisition of Knudra Transgenics by InVivo Biosystems, raised over $5M in venture capital and $7M in non-dilutive SBIR funding, licensed CRISPR technology in 2014 — shortly after it was inve...more
Why Do All Daughters of an Affected Father Inherit Fabry Disease? — DECODE No. 11
(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
