Redefining Endpoints Through Lived Experience: Jon Hagstrom’s Story of Patient Advocacy in Alpha-1 Antitrypsin Deficiency

A1F Board Chair Jon Hagstrom shares with the Critical Path Institute (C-Path) how his Alpha-1 journey is informing research and […]

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‘Substantially underdiagnosed’: Expert consensus moves to redefine rare liver disease 

“The continuation of achieving our goals hinges on detecting, testing, finding and helping every Alpha. We have increased our efforts […]

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FDA Launches Framework for Accelerating Development of Individualized Therapies for Ultra-Rare Diseases

FDA NEWS RELEASE The U.S. Food and Drug Administration today issued draft guidance for sponsors seeking approval for targeted individualized […]

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Wave Life Sciences Announces Plans to Accelerate Regulatory Engagement with Full Control of WVE-006 for Alpha-1 Antitrypsin Deficiency

“We are thrilled with Wave Life Sciences commitment to the Alpha-1 patient community. Their acceleration of regulatory engagement of the […]

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Tessera Therapeutics Announces FDA Clearance of IND Application for its Lead In Vivo Gene Editing Program TSRA-196 for AATD

PRESS RELEASE SOMERVILLE, Mass., January 12, 2026 /GLOBE NEWSWIRE/ Tessera Therapeutics, the biotechnology company pioneering a new approach in genetic […]

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Alpha-1 Global Meeting held in Argentina 

The second Latin American Alfa-1 Meeting titled “Consolidando avances, ampliando horizontes”, (“Consolidating Advances, Expanding Horizons”) was held on December 10, 2025, in Buenos Aires, Argentina.  Organized by Alfa1 Argentina and supported by […]

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Kamada Announces Discontinuation of its Phase 3 Inhaled AAT Clinical Trial

“Thank you to the Alpha-1 patients that participated in this important clinical trial, InnovAATe. It is because of the dedication of patients […]

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