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.

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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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