Welcome to the Rare Classroom, a new series from Patient Worthy. Rare Classroom is designed for the curious reader who wants to get informed on some of the rarest, most…
Ryner Lai, MBBS, a medical graduate of Queen Mary, London University, is a physician and writer with a passion to use medical research as a way of improving patients’…
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…
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…
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…
According to a recent article, a cyclist who was closely affected by alpha-1 antitrypsin deficiency (A1AD), is biking across Australia with his good friend to raise awareness and money for…
According to a recent article, a Vietnam veteran is spreading awareness about the struggles of accessibility for treatments for his rare genetic disease alpha-1 antitrypsin deficiency (A1AD). Alpha-1 Antitrypsin Deficiency…
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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…
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…
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…
According to an article published in the Orphanet Journal of Rare Diseases, a workshop held on June 23, 2019 in Orlando, FL focused on a difficult subject: improving detection and diagnosis for…
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…
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…
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