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Why Rare Diseases Like Fabry Stay Underfunded by Big Pharma
Glafabra
A pharma company will almost always choose a blockbuster over a small patient population. That's not cynicism — it's how the economics of a multibillion-dollar portfolio actually work.
It's also exactly why rare diseases like Fabry, Pompe, and Gaucher stay underserved relative to their severity, and why community funding models — ours included — exist at all. A therapy addressing a few thousand pe...more
Why Fabry Disease Requires a Multidisciplinary Care Team
Fabry disease touches the kidneys, heart, nerves, skin, and GI tract — so no single specialist manages it alone.
Patients typically see a rotating team: a metabolic or genetics specialist, a nephrologist, a cardiologist, a neurologist, sometimes a pain specialist. Coordinating all of that is its own quiet burden on patients and families.
Glafabra's own team mirrors that same instinct toward breadt...more
Why AAV Gene Therapy for Fabry Disease Can't Be Repeated
The AAV-waned patient population isn't hypothetical anymore — it's showing up in Glafabra's inbox.
Every AAV gene therapy for Fabry disease triggers a permanent neutralizing antibody response after the first dose, so when expression eventually fades — usually three to five years in — there's no way to re-treat with that same modality. One patient enrolled in an AAV trial has already been referred ...more
Designing One Clinical Trial for Two Regulators: FDA and PMDA
Designing a trial for two regulators at once is harder than designing it for one — but it's cheaper than doing it twice.
Glafabra's U.S. Phase 1/2 protocol is being built from the outset to generate the specific biomarker package — alpha-Gal A enzyme activity, plasma lyso-Gb3, eGFR slope — that PMDA would require for a Japan submission. That's a deliberate choice made now, months before IND filing...more
Meet Glafabra's Acting CFO, Sidney Norton
Meet Sidney (Sid) Norton, Acting Chief Financial Officer.
Sid brings 25 years across private, government, and not-for-profit healthcare finance. He currently serves as CFO of Intermountain Primary Children's Hospitals, and previously held senior financial and operational roles at Cincinnati Children's, including VP of the Cancer and Blood Diseases Institute, plus five years in system finance at In...more
What Percentage of Fabry Patients Can Take Oral Therapy? — DECODE No. 15
(Answer to the question last time on August 20: lupus, multiple sclerosis, and other conditions with overlapping symptoms are common Fabry misdiagnoses.)
There's an oral therapy for Fabry disease, but it only works for patients whose specific GLA variant responds to it.
Roughly what percentage of Fabry patients carry a variant that qualifies for oral therapy? Drop your guess in the comments below.
...more
Six Weeks Into Testing the Waters: Glafabra's Progress Update
Six weeks into Testing the Waters. Here's where things actually stand.
FDA INTERACT meeting: done. IND protocol: in development. University of Utah Health: planned to be the primary clinical site. DECODE has answered 13 real questions about Fabry disease so far, and our LinkedIn Tuesdays are now team introductions.
If you've been reading since Day 1 — thank you. If you just found us — welcome, and...more
Ask Glafabra Anything: Live Q&A on the BioTech Funding Portal
This portal is a live Q&A, not a static page.
Questions we've gotten recently: Why did Glafabra choose melphalan over busulfan for conditioning? What happens to the corrected cells over the long term? Why is University of Utah Health planned to be the clinical site?
We answer every one publicly so the whole community benefits from the answer, not just the person who asked.
What's your question...more
