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Endpoints·3h ago·1 min read
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FDA approves Mirum's Atebrioz for ultra-rare disease that hardens muscle and connective tissue

Sep 25, 2026·read at Endpoints ↗ApprovalPositive

The U.S. Food and Drug Administration has granted approval for Mirum Pharmaceuticals' new therapy for an ultra-rare genetic disorder that causes muscle and connective tissue to turn into bone.

The drug, zilurgisertib, will be sold under the brand name Atebrioz and works by blocking the ALK2 receptor, a key driver of the disease process.

This is the third approved treatment for the condition, providing an additional option for patients facing progressive heterotopic ossification.

source

This writeup was produced by pharmadog from original reporting by Endpoints.

Original headline: “FDA approves Mirum’s drug for ultra-rare soft tissue disease”

read at Endpoints ↗
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companies & drugs in this story

companies
MIRUM5Ultragenyx Pharmaceutical Inc.3
topics
Orthopedics3Rare Disease3Approval

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