Stay informed about breakthrough developments, policy changes, and community news that are transforming the landscape of Alpha-1 Antitrypsin Deficiency diagnosis, treatment, and care.
Breaking News: Kamada Ltd. announced the discontinuation of its Phase 3 InnovAATe clinical trial studying inhaled Alpha-1 Antitrypsin therapy.
What This Means:
While disappointing, this development highlights the ongoing challenges in developing alternative AAT delivery methods. The Alpha-1 Foundation thanked the patients who participated in this important clinical trial, emphasizing that their dedication advances the field even when trials don't achieve their primary endpoints.
Our Take: This underscores the continued need for research into optimal AAT formulations and delivery methods. The Mark Egly Foundation supports diverse approaches to making AAT therapy more effective and accessible.
🔗 Read the Alpha-1 Foundation announcement
Expanding Access: The University of Kentucky has been officially designated as a Clinical Resource Center (CRC) by the Alpha-1 Foundation, expanding the network of specialized AATD care facilities.
Significance: Clinical Resource Centers provide comprehensive, multidisciplinary care for individuals with AATD, bringing together pulmonologists, hepatologists, genetic counselors, and other specialists. This designation improves access to expert care for patients in Kentucky and surrounding regions.
Impact: More CRCs mean better access to diagnosis, treatment, and specialized care—exactly what the Mark Egly Foundation advocates for in changing standards of care.
🔗 Learn more about the UK CRC designation
Global Collaboration: The second Latin American Alfa-1 Meeting, titled "Consolidating Advances, Expanding Horizons," was held in Buenos Aires, Argentina, bringing together clinicians, researchers, and patient advocates from across Latin America.
Why It Matters: AATD is a global issue requiring international collaboration. This meeting demonstrates growing awareness and improved infrastructure for AATD diagnosis and treatment in Latin America—regions that have historically had limited access to specialized care.
🔗 Read about the Argentina meeting
Empowering Patient Voices: The Alpha-1 Foundation launched the Fred C. Walsh Speaker Bureau to amplify patient voices and elevate patient-centered work in research, policy, and healthcare delivery.
Connection to Our Mission: Patient advocacy is central to changing standards of care. When patients share their stories with physicians, policymakers, and the public, they drive awareness and systemic change. The Mark Egly Foundation strongly supports patient-led advocacy efforts.
According to ClinicalTrials.gov, there are currently 289 clinical studies related to Alpha-1 Antitrypsin registered globally. These studies span:
Gene Therapy
Novel Augmentation Therapies
Expanded Indications
Biomarker Development
🔗 Search Alpha-1 Antitrypsin clinical trials
The Alpha-1 Foundation continues to expand its global leadership role, recently highlighting its strategic engagement with the European Union to improve AATD awareness, diagnosis, and treatment across Europe.
Policy Priorities:
What We're Watching:
🔗 Read about EU strategic engagement
The Mark Egly Foundation is developing a comprehensive program to transform how patients and families navigate Alpha-1 Antitrypsin Deficiency:
Early Identification
Genetic Counseling
Treatment Navigation
Comprehensive Care Coordination
Clinical Trial Matching
Patient Education Resources
Family Education
Healthcare Provider Education
Peer Support Networks
Advocacy Training
Events and Engagement
Alpha-1 Research Registry
Clinical Trial Opportunities
The Alpha-1 Foundation provides monthly updates on peer-reviewed research publications funded by their research program. Recent publications cover:
🔗 View November PubMed articles
1. Physician Education Campaign
2. Medical School Curriculum Integration
3. Insurance Coverage Advocacy
4. Clinical Guideline Evolution
5. Alternative Production Research
November 2025 marked another successful Alpha-1 Awareness Month, with the Alpha-1 Foundation celebrating its 30th anniversary. The community came together to:
Impact: Awareness campaigns are crucial for earlier diagnosis. Every person who learns about AATD becomes a potential advocate who might recognize symptoms in themselves or others.
🔗 Read Week 4 and Week 5 recaps
The Alpha-1 community mourns the loss of Dr. Gordon Cadwgan, PhD, who passed away peacefully on November 29, 2025, at 81 years old. Dr. Cadwgan was a dedicated researcher and advocate whose work advanced the understanding and treatment of AATD.
His legacy reminds us why this work matters and why we must continue the fight until everyone affected by AATD has access to diagnosis, treatment, and hope.
January 2026: Virtual Education Day & State of the Union
The Alpha-1 Foundation will host its 2026 Virtual Education Day and State of the Union on January 28, 2026, providing updates on research, advocacy, and community initiatives.
🔗 Register for the 2026 Virtual Event
Upcoming Research Milestones
Policy Developments
For Patients:
For Healthcare Providers:
For Everyone:
Mark Egly Foundation
Alpha-1 Foundation Resources
The standard of care is evolving. Stay informed, stay engaged, and be part of the transformation.
Together, we're building a future where Alpha-1 Antitrypsin Deficiency is diagnosed early, treated effectively, and understood comprehensively—changing lives and saving families from preventable suffering.