(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.

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

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

FDA’s written feedback generally noted that the nonclinical package supports a proposed exploratory clinical study, recommended prioritizing plasma lyso-Gb3 as the pharmacodynamic readout, agreed that assessing eGFR is reasonable while recommending at least two years of follow-up, said a within-subject exploratory design is reasonable, and advised that manufacturing knowledge may be leveraged acro...more

Same-day discharge isn't a footnote. For a patient and their family, it's the difference between one afternoon and a hospital stay.

Four of five FACTS trial patients received their conditioning regimen and went home the same day — because it used a gentler, non-myeloablative approach instead of bone-marrow-transplant-intensity chemotherapy. Glafabra built Live-cel around that outpatient approach f...more

(Answer to the question last time on August 17: women — X-inactivation can leave female patients with near-normal enzyme levels on a standard blood test, so genetic testing of the GLA gene is the reliable way to diagnose them.)

Fabry's symptoms touch so many organ systems that it regularly gets mistaken for other conditions entirely — sometimes for years.

Name one condition Fabry disease is common...more

Six weeks out from two Japan milestones.

October 2: IFFD 2026, the international Fabry symposium at Jikei University in Tokyo — where Glafabra co-founder Dr. Jeff Medin will present Live-cel data to the global Fabry specialist community.

October 7–9: BIO Japan in Yokohama, where the team meets Japan pharma partners directly.

As Director of APAC Strategies, this is the stretch of the calendar I've ...more