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Which Enzyme Is Missing in Fabry Disease? — DECODE No. 1
I'm a single enzyme. When I go missing, a fatty molecule quietly piles up inside your cells — silently, for years — until it starts to damage kidneys, heart, and nerves.
My name begins with the first letter of the Greek alphabet.
Who am I? Drop your guess in the comments — the answer posts with DECODE No. 2.
Follow the whole series: https://b2f.bz/5k9n3
@Brian Christie @Neva W...more
LSDs + protein deficiency: a $13B Market and Rising
The Fabry Market Is Bigger Than the Headlines, and the Platform Behind It Is Bigger Still
Every few months a new acquisition reminds the market that lysosomal storage disorders are quietly one of the most durable franchises in rare disease. BioMarin's $4.8B acquisition of Amicus in April 2026 was the latest. So it is worth doing the arithmetic out loud, from primary sources, on exactly how large t...more
Glafabra Therapeutics Receives FDA Acceptance for Face-to-Face INTERACT Meeting for GT-GLA-S03, Its Cell-Based Gene Therapy for Fabry Disease
Glafabra granted a face-to-face INTERACT meeting slated to occur July 16, 2026.
We will be discussing with the FDA key questions that will set the development path of our GT-GLA-S03 cell-based gene therapy for Fabry in its journey towards the market.
This milestone achievement was only made possible with the amazing contributions of the internal team and our contractors. Many thanks to Elizabeth W
