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.


First Gene Therapy for an Inherited Disease Gets Approved by the FDA

The Food and Drug Administration (FDA) approved the nation’s first gene therapy for an inherited disease, a treatment that improves the sight of patients with …

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Organovo Receives Orphan Designation From U.S. FDA for 3D Bioprinted Therapeutic Liver Tissue Treatment of Alpha-1 Antitrypsin Deficiency

SAN DIEGO, Dec. 26, 2017 (GLOBE NEWSWIRE) — Organovo Holdings, Inc.(NASDAQ:ONVO) (“Organovo”) today announced that the U.S. Food and Drug Administration (“FDA”) granted orphan drug designation for the Company’s treatment of …

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Arrowhead Pharmaceuticals Files for Regulatory Clearance to Begin Phase 1 Study of ARO-AAT

(December 20, 2017 07:30 AM Eastern Standard Time)   PASADENA, Calif.–(BUSINESS WIRE)–Arrowhead Pharmaceuticals Inc. (NASDAQ: ARWR) today announced that it filed a regulatory submission to …

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Adverum Biotechnologies Doses First Patient in the ADVANCE Phase 1/2 Clinical Trial of ADVM-043 for A1AT Deficiency

Company Expects to Report Preliminary Data in the Second Half of 2018   MENLO PARK, Calif., Dec. 28, 2017 (GLOBE NEWSWIRE) — Adverum Biotechnologies, Inc. (Nasdaq:ADVM), a clinical-stage gene …

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ATS Renames PAR Excellence Award After John W. Walsh

The American Thoracic Society (ATS) Public Advisory Roundtable was moved to rename the annual PAR Excellence Award by a unanimous vote in November. The award, …

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HSE Covers Administration Costs of Therapy for Irish Alpha-1 Patients After Second Alpha Dies

By Catherine Shanahan Health Correspondent Irish Examiner December 04, 2017 The HSE has agreed to cover the administration costs of a therapy known to slow …

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Alpha-1 Patient Action Group Mounts Vigil at HSE HQ as Stand-off Continues Between CSL Behring and HSE Over Provision of Therapy to Clinical Trial Patients

CSL Behring & HSE to meet today to discuss situation, as patients urge both sides to find a way forward. Date of issue: 16th November, …

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UK Company Acquires License for Potential Pill-based Alpha–1 Lung Therapy

Mereo BioPharma Group, a clinical stage, UK-based, biopharmaceutical company focused on rare and specialty diseases, announced that it has reached an agreement with AstraZeneca for …

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New Gene Editing Approach for Alpha-1 Antitrypsin Deficiency Shows Promise in UMMS Study

A new study by scientists at UMass Medical School shows that using a technique called “nuclease-free” gene editing to correct cells with the mutation that …

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Arrowhead Presents Promising Preclinical Data on Development of ARO-AAT for Treatment of Alpha-1 Liver Disease

Data Presented in Late Breaking Poster Company Expects to File Clinical Trial Application in First Quarter 2018 to Begin First-in-Human Studies of ARO-AAT (PASADENA, CA, …

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Alpha-1 Around The Globe

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