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

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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Enrollment Has Been Completed For a Clinical Trial of ARO-AAT, an Investigational Therapy for 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…

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

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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