According to a late October 2021 news release from biopharmaceutical company Mereo BioPharma Group plc ("Mereo"), the company's therapeutic candidate alvelestat received Orphan Drug designation from the FDA. The therapy…
According to a relatively recent news release from biopharmaceutical company Dicerna Pharmaceuticals, Inc. ("Dicerna"), interim data is now available from a Phase 1 clinical trial evaluating belcesiran for patients with…
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…
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…
About six months ago, pharmaceutical company Takeda pledged $300M towards developing Arrowhead Pharmaceuticals' ("Arrowhead") ARO-AAT, an RNA-silencing treatment for patients with alpha-1 antitrypsin deficiency (A1AD). According to Fierce Biotech, the…
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…
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…
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…
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…
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…
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…
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…
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…
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…
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…
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…
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…
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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