Discerna Pharmaceuticals has just announced the results from a Phase 1 trial investigating their GalXC™ RNAi therapy (Belcesiran) for alpha-1 antitrypsin (AAT) deficiency-associated liver disease (AATLD). The results demonstrated both…
A retrospective medical records study for interstitial lung disease (ILD) has just been published in Arthritis Care & Research. The results found that for women diagnosed with ILD as well as an…
A new study has demonstrated that plasma desmosine could be an acute aortic syndrome biomarker. This also means it could be a potential diagnostic tool. The findings from this study…
Fatty Liver Disease This condition is exactly what it sounds like- excess fat accumulates in the liver and causes damage. The condition can have many negative consequences on the body.…
Equillium Inc. has just announced that their most recent study in lupus nephritis (LN) has been published in the Journal of Clinical Investigation. This study is thanks to collaboration with the Accelerated Medicines…
Albireo Pharma Inc. has just announced the results from their Phase 1 clinical trial for A3907, an ASBT inhibitor. The study produced positive results, achieving its primary and secondary endpoints. Researchers…
The FDA has just announced its accelerated approval of the very first therapy which can reduce proteinuria (high levels of protein in the urine) in patients diagnosed with primary immunoglobulin…
A study conducted by Rayyan Zafar and his colleagues from the Imperial College London was recently published in BMJ Paediatrics Open. This investigation evaluated the efficacy of whole-plant cannabis as a treatment for…
Givlaari, a treatment for acute hepatic porphyria (AHP), was first approved in Europe back in March of 2020. However, approval doesn't mean immediate access unfortunately. It's been nearly two years…
Dr. Joshua Sabari, a professor at New York University's Department of Medicine, has recently spoken about the different treatment options for ALK-positive non-small cell lung cancer (NSCLC). Specifically, he discussed…
Non-clear cell renal cell carcinoma (nccRCC) has recently been found to have a very strong connection with various subtypes of proliferative disease. Further, this condition was found to have a…
Dr. Deborah M. Stephens from the Huntsman Cancer Institute recently presented at the 39th Annual Chemotherapy Foundation Symposium Innovative Cancer Therapy for Tomorrow (CFS). The presentation surrounded how critical identifying…
Krystal Biotech has just announced results from their Phase 3 trial called GEM-3, and the results were all positive! This trial investigated bermemagene geperpavec (B-VEC), or VYJUVEKTM as a potential therapy…
Cancer recurrence as a result of drug resistance has been an ongoing battle. Far too many patients start treatment, experience great results, and then suddenly have their tumor reappear. Quickly…
Doctor Jeffrey Miner, who is a member of the Alport Syndrome Foundation's (ASF) Scientific Advisory Research Network, was just awarded a Ro1 grant worth 2.25 million dollars for his research in Alport…
A new method to differentiate between two gynecologic tumors, malignant epithelial ovarian tumors (MEOTs) and borderline epithelial ovarian tumors (BEOTs), has been created by Chinese scientists from the Suzhou Institute…
CONTENT WARNING: Severe skin wounds and damage Working in rare disease is always hard- It means working in suffering and grief, but even with all our experience, we had to…
Even as COVID-19 cases die down, post-COVID syndrome still plagues many individuals across the world. These individuals have faced ongoing COVID-19 symptoms for more than two weeks following diagnosis. The…
Soligenix Inc. has just announced that they've received a Pediatric Investigation Plan waiver, or PIP waiver from the MHRA, the UK's marketing agency. Now, Soligenix Inc has a pediatric waiver…
GlaxoSmithKline has recently announced their new approval of Nucala by the European Commission. This therapy has been approved for three new diseases, all of which are driven by eosinophils. Nucala is…
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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…
A recent clinical trial called CREDENCE has uncovered that canagliflozin, a sodium-glucose cotransporter 2 (SGLT2), can reduce hyperkalemia risk in chronic kidney disease (CKD) and type 2 diabetes patients who…
A recent study has found that the combination of temozolomide (T), vincristine (V), and irinotecan (I), may be the best standard of care for all patients (children and adults) diagnosed…
The National Institute for Health and Care Excellence (NICE) has just recommended a gene silencing treatment for acute hepatic porphyria (AHP), a rare disease. AHP AHP is a term for…
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…
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