Alpha 1 Global

Our mission is to develop a collaborative global network of Alpha-1 patient leaders, physicians, and researchers, to increase awareness, detection, and access to care for Alphas around the world.


Orphan Drug Designation Granted to Bronchiolitis Obliterans Treatment

Onspira Therapeutics announced that the U.S. Food and Drug Administration (FDA) granted Orphan Drug Designation to its investigational product, OSP-101, for the treatment of Bronchiolitis …

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Alliance for Regenerative Medicine Publishes Position on Proposed Regulation on HTAs and Amending Directive 2011/24/EU

BRUSSELS, Belgium and WASHINGTON, D.C. – July 11, 2018 The Alliance for Regenerative Medicine (ARM), the international advocacy organization representing multiple stakeholder groups comprising the …

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Alpha-1 Foundation Board has New Chair

The Alpha-1 Foundation has the honor to announce that Jeanine D’Armiento, MD, PhD, has been named as the newest Chair of its board of directors. …

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Inhaled Alpha-1 Antitrypsin: Kamada Receives Positive Advice on New Phase 3 Study Design

Kamada Ltd., a plasma-derived protein therapeutics company, announced that it has received positive scientific advice from the Committee for Medicinal Products for Human Use (CHMP) …

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New Working Group for Patient Advocacy is Created

The Federal Drug Administration (FDA) and the Clinical Trials Transformation Initiative (CTTI) announced newly selected representatives for the Patient Engagement Collaborative (PEC), a joint endeavor …

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CRISPR Gene Editing May Correct Alpha-1 But Perhaps It Is Too Soon

Groundbreaking research shows that CRISPR-Cas9 genome editing technology can correct the gene mutation responsible for Alpha-1 Antitrypsin Deficiency (Alpha-1), by successfully making a targeted gene …

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The 27th National Education Conference Opens with a Panel on the Latest Alpha-1 Findings

The 27th Annual Alpha-1 National Education Conference, organized by the Alpha-1 Foundation in San Francisco, California, will open this Friday, June 29th with a panel …

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Arrowhead Completes Enrollment in Phase 1 Study for Treatment of Alpha-1 Liver Disease

Arrowhead Pharmaceuticals, Inc. announced that it has completed enrollment of a Phase 1 clinical trial of ARO-AAT, their second generation subcutaneously administered RNA interference (RNAi) …

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RNA Interference Therapies Could be on the Verge of Success

In the mid-2000s there was a wave of excitement across the pharmaceutical industry: a new discovery about cells that had the potential to transform medicine. …

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Consortium Seeks Common Standards for Gene-editing Therapies

How to best measure if a therapy is reaching its target gene without introducing errors in others? This is one of the big questions that …

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Alpha-1 Around The Globe

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