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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Gene Therapy Meeting Draws Record Numbers to Make a Difference
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Gene Therapy Meeting Draws Record Numbers to Make a Difference

According to a report by MedCityNews, gene therapies have been catching increasing interest. They’ve been popular in the news and hot talk among medical professionals, but it seems like the…

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

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
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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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A 3-Step Guide to Help You Move Forward After an Alpha-1 Diagnosis
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A 3-Step Guide to Help You Move Forward After an Alpha-1 Diagnosis

The Alpha-1 Foundation has a website dedicated to Alpha-1 patients and caregivers. It offers a wealth of information and resources about about the disease. Alpha-1 Antitrypsin Deficiency (Alpha-1) is a…

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Online Course Provides Information on Alpha-1 Antitrypsin Deficiency
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Online Course Provides Information on Alpha-1 Antitrypsin Deficiency

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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Coming Soon: Phase III Trial For Inhaled Alpha-1 Antitrypsin Therapy
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Coming Soon: Phase III Trial For Inhaled Alpha-1 Antitrypsin Therapy

The pharmaceutical company, Kamada, has just submitted its proposed Phase III protocol for an inhaled alpha-1 antitrypsin therapy (inhaled AAT) to the U.S. Food and Drug Administration (FDA). This therapy…

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

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

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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