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LSDs + protein deficiency: a $13B Market and Rising

Chris Hopkins Chris Hopkins
Posted at Jun 21

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

Categories: Fabry Disease
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Glafabra Therapeutics Receives FDA Acceptance for Face-to-Face INTERACT Meeting for GT-GLA-S03, Its Cell-Based Gene Therapy for Fabry Disease

Chris Hopkins Chris Hopkins
Posted at May 18

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

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Categories: Fabry Disease
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