For more than 20 years, I had a treatment routine that worked. My specialty pharmacy delivered the medications I needed and coordinated trained nurses who came to my home for…
Sanofi has announced encouraging results from a Phase 2 clinical trial of efdoralprin alfa, an investigational therapy for alpha-1 antitrypsin deficiency (AATD), a rare genetic disorder affecting the liver and…
Sanofi has announced encouraging results from its Phase 2 ElevAATe clinical trial evaluating efdoralprin alfa (SAR447537), a recombinant human alpha-1 antitrypsin (AAT)-Fc fusion protein, for the treatment of emphysema caused…
The Rady Children’s Institute for Genomic Medicine (RCIGM) has announced a landmark international collaboration with Sidra Medicine in Qatar to launch the BeginNGS® newborn genome sequencing program—marking a significant step…
The Alpha-1 Foundation (A1F) has announced a groundbreaking advancement in the fight against Alpha-1 Antitrypsin Deficiency (AATD) with the launch of AlphaDetect, a dedicated non-profit subsidiary focused on revolutionizing the…
Clinical studies are critical tools in identifying and developing therapeutics for a wide variety of conditions. This research is especially important in the rare disease and chronic illness spaces,…
According to an a press release published on Business Wire, Arrowhead Pharmaceuticals Inc. recently presented updated phase 2 data at the 2023 European Association for the Study of the Liver…
According to a story from globenewswire.com, the biopharmaceutical company Mereo BioPharma Group plc has recently announced that the company's investigational therapeutic alvelestat has earned Fast Track designation from the US…
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…
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…
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…
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…
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…
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…
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