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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
Cultivating an ecosystem of trust through principled decision-making
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
Investor Education Resources Now Available
We’ve published a comprehensive set of investor education materials to help participants better understand how Regulation Crowdfunding works on the BioTech Funding Portal. These resources are intended to explain the mechanics of crowdfunding offerings, the role of a registered funding portal, and the regulatory framework governing issuers and investors. Whether you are new to crowdfunding or revie...more
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