Results From This Study are Encouraging for Alpha-1 Antitrypsin Deficiency Liver Disease
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Results From This Study are Encouraging for Alpha-1 Antitrypsin Deficiency Liver Disease

  Takeda and its collaborator, Arrowhead Pharmaceuticals, recently announced results from AROAAT-2002 of the investigational drug fazirsiran. The drug is an RNA interference therapeutic developed to lessen alpha-1 antitirypsin protein…

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Positive Trial Results Released for Alpha-1 Antitrypsin Deficiency-Associated Emphysema Treatment

Mereo BioPharma Group has just announced positive results from their Phase 2 study called ASTRAEUS. This study investigated alvelestat (MPH-966), which is an oral neutrophil elastase inhibitor, as a treatment…

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Patient Stories: A1AD Patients Reunite with Transplant Surgeon
Advancements in transplant surgery has given cystic fibrosis patients new hope for successful lung transplants.

Patient Stories: A1AD Patients Reunite with Transplant Surgeon

Many father-son duos are close, but this pairing - reported on in an article from CBS58 - shares a special bond that was strengthen by the shared experience of a…

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The Fight For Access For Alpha-1 Antitrypsin Deficiency Treatment Across Canada

November was alpha-1 antitrypsin deficiency (A1AD) awareness month, and A1AD is certainly a rare disease which deserves more awareness. Even with universal healthcare in Canada, many patients who have alpha-1 antitrypsin deficiency…

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New Treatment For Alpha-1 Antitrypsin Deficiency Lung Disease Has Been Granted Orphan Drug Designation

Mereo BioPharma Group has just announced that they have received Orphan Drug Designation from the FDA for their new treatment for alpha-1 antitrypsin deficiency (A1AD) called alvelestat. This therapy is…

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Promising Data from Phase 2 VX-864 Study for A1AD, Despite Discontinuation
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Promising Data from Phase 2 VX-864 Study for A1AD, Despite Discontinuation

Medical studies are crucial players in the journey to develop new and more efficacious treatment options for patients with rare diseases. On June 10, 2021, Vertex Pharmaceuticals Incorporated ("Vertex") shared…

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Study Finds Alpha-1 Antitrypsin Deficiency Could Benefit From Base Editing
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Study Finds Alpha-1 Antitrypsin Deficiency Could Benefit From Base Editing

Researchers from Boston University and Boston Medical Center have recently uncovered that base editing is able to correct the gene mutation which causes alpha-1 antitrypsin deficiency (A1AD). This condition impacts…

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Baylor College Team Discovers the Origin of Alpha-1 Antitrypsin Deficiency in Infants
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Baylor College Team Discovers the Origin of Alpha-1 Antitrypsin Deficiency in Infants

  A recent article in Science News highlighted a discovery by Richard Seifes, M.D. of Baylor College of Medicine.  Dr. Seifes has discovered the origin of a liver disease that…

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New Collaboration Will Develop ARO-AAT for A1AD-Related Liver Disease
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New Collaboration Will Develop ARO-AAT for A1AD-Related Liver Disease

  In October 2020, Arrowhead Pharmaceuticals ("Arrowhead") announced a new collaboration and licensing agreement with global biopharmaceutical company Takeda Pharmaceutical Company Limited ("Takeda"). Together, the pair will develop and commercialize…

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

  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
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Alpha-1 Antitrypsin Deficiency Treatment Receives Orphan Drug Designation

  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
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The Relationship Between Asthma and 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
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FDA Grants Orphan Drug Designation for DCR-A1AT for Treatment of 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

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

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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New Phase 2/3 Clinical Trial for Liver Disease in Alpha-1 Antitrypsin Deficiency Should be Coming Soon

Alpha-1 Antitrypsin Deficiency Alpha-1 Antitrypsin Deficiency (AATD) is a rare disease caused by low levels of the Alpha-1 Antitrysin protein. It can lead to lung disease and liver disease. Liver…

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