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Patients with Alpha-1 Antitrypsin Deficiency Liver Disease Show Improvement After Six Months of Experimental Treatment

• Post author:Rose Duesterwald
• Post published:September 29, 2020
• Post category:Alpha-1 Antitrypsin Deficiency/Rare Disease

  Arrowhead Pharmaceuticals recently announced positive twenty-four-week biopsy results from four patients who participated in the first cohort of the Phase II clinical trial of ARO-AAT. ARO-AAT is Arrowhead’s investigational…

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Alpha-1 Antitrypsin Deficiency Treatment Receives Orphan Drug Designation

• Post author:Jessica Lynn
• Post published:March 31, 2020
• Post category:Alpha-1 Antitrypsin Deficiency/Hepatocellular Carcinoma

  In June 2019, Dicerna Pharmaceuticals submitted a clinical trial application for their DCR-A1AT therapy and expects to treat the first patient with A1AD-related liver disease near the end of…

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The Relationship Between Asthma and Alpha-1 Antitrypsin Deficiency

• Post author:Kendall Mason
• Post published:January 6, 2020
• Post category:Alpha-1 Antitrypsin Deficiency

  Alpha-1 antitrypsin deficiency (A1AD) shares similarities with asthma, which leads to it often being misdiagnosed as asthma. This misdiagnosis leads to insufficient treatment. Toronto Western Hospital's Asthma and Airways…

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FDA Grants Orphan Drug Designation for DCR-A1AT for Treatment of Alpha-1 Antitrypsin Deficiency

• Post author:Kendall Mason
• Post published:December 20, 2019
• Post category:Alpha-1 Antitrypsin Deficiency

Dicerna Pharmaceuticals works in ribonucleic interference (RNAi) therapies as treatments of rare diseases. They have recently been granted the orphan drug designation by the European Commission for their drug, DCR-A1AT.…

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New Clinical Trial Application for Alpha-1 Antitrypsin Deficiency-Associated Liver Disease Submitted in Sweden

• Post author:Trudy Horsting
• Post published:July 3, 2019
• Post category:Alpha-1 Antitrypsin Deficiency

Dicerna Pharmaceuticals has just announced that they have submitted a Clinical Trial Authorization application for their investigational therapy DCR-A1AT. This application has been submitted to the Swedish Medical Products Agency…

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The Alpha-1 Foundation’s Alpha-1 National Conference

• Post author:James Moore
• Post published:June 21, 2019
• Post category:

The 28th Annual Alpha-1 National Conference Orlando, Florida Are you an alpha-1 antitrypsin deficiency patient? Well don’t miss this important patient event. At this conference, alpha-1 antitrypsin deficiency patients and…

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Animal Model Study Using CRISPR Could Lead to Successful Treatment of Rare Lung Diseases

• Post author:James Moore
• Post published:April 23, 2019
• Post category:Alpha-1 Antitrypsin Deficiency/Cystic Fibrosis/surfactant deficiency

According to a story from EurekAlert!, a group of researchers affiliated with Penn Medicine and Children's Hospital of Philadelphia recently conducted an experiment in which the scientists used CRISPR gene…

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A New Study Explores Future Directions for Alpha-1 Antitrypsin Deficiency Treatment and Research

• Post author:Anna Hewitt
• Post published:July 18, 2018
• Post category:Alpha-1 Antitrypsin Deficiency

A recent review, carried out by researchers from Spain and published in the Orphanet Journal of Rare Diseases (OJRD), discusses the rare condition alpha-1 antitrypsin deficiency. The original study, which…

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The Alpha-1 Foundation’s Annual National Education Conference

• Post author:James Moore
• Post published:June 29, 2018
• Post category:

27th Annual Alpha-1 National Education Conference This event will allow Alpha-1 and COPD patient families to learn more about their diseases and stay informed about the latest in research and…

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Enrollment Has Been Completed For a Clinical Trial of ARO-AAT, an Investigational Therapy for Alpha-1 Antitrypsin Deficiency

• Post author:Anna Hewitt
• Post published:June 25, 2018
• Post category:Alpha-1 Antitrypsin Deficiency

Arrowhead Pharmaceuticals, Inc. have announced that they have completed patient enrollment for a Phase 1 clinical trial investigating ARO-AAT, an investigational therapy for the treatment of a genetic liver disease…

Continue Reading Enrollment Has Been Completed For a Clinical Trial of ARO-AAT, an Investigational Therapy for Alpha-1 Antitrypsin Deficiency

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A Genetic Test for Alpha-1 Antitrypsin Deficiency Gets Approval from the FDA

• Post author:James Moore
• Post published:February 8, 2018
• Post category:Alpha-1 Antitrypsin Deficiency

According to an article at Clinical Lab Products, a new genetic test for alpha-1 antitrypsin deficiency was recently approved by the Food and Drug Administration (FDA). This certification is the…

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Bioprinted Liver Tissue Could Help Treat A1AD, And it Just Got Orphan Drug Status

• Post author:James Moore
• Post published:January 10, 2018
• Post category:Alpha-1 Antitrypsin Deficiency/Rare Disease

Organovo Holdings recently stated that its 3D bioprinted therapeutic liver tissue was granted orphan drug status by the Food and Drug Administration (FDA). Taylor Crouch, the company CEO, was excited…

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Online Course Provides Information on Alpha-1 Antitrypsin Deficiency

• Post author:Lyssé Morganette
• Post published:September 19, 2017
• Post category:Alpha-1 Antitrypsin Deficiency/Rare Disease

If you or someone you love has bronchiectasis or COPD, you should be aware of the risk of alpha-1 antitrypsin deficiency, or A1ATD. Alpha-1 antitrypsin disorder is an inherited condition…

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A Wasp Lead This Dancer to Her Alpha-1 Antitrypsin Deficiency Diagnosis

• Post author:Trudy Horsting
• Post published:June 29, 2017
• Post category:Alpha-1 Antitrypsin Deficiency/Rare Disease

According to the Daily Mail, Clare Fray has alpha-1 antitrypsin deficiency. But for years she thought her symptoms were just a part of normal life. She suffered from numerous chest…

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Kill Thousands of Birds With One Stone Using This Alpha-1 Antitrypsin Deficiency Resource

• Post author:Trudy Horsting
• Post published:June 27, 2017
• Post category:Alpha-1 Antitrypsin Deficiency/Rare Disease

The goal of the Alpha-1 Foundation is ultimately to find a cure for Alpha-1 Antitrypsin Deficiency (A1AD). But along the way, they are also a resource for patients, families, and…

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