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.…
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…
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…
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…
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…
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…
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…
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…
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…
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…
For patients, caregivers, healthcare providers, and anyone interested in learning more about liver diseases, there is a website called Focus. This website is organized by CLDF, which stands for Childhood…
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…
Ron Taylor is a smoker who has the lungs of a 126-year-old man. According to the Alpha-1 Foundation's website and the Memorial University Gazette, his diagnosis started when he went…
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…
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…
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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