According to a story from GlobeNewswire, the biopharmaceutical company Immunomedics, Inc., has recently announced that its medication sacituzumab govitecan-hziy (marketed as Trodelvy) has recently earned Orphan Drug designation from the…
Researchers have just uncovered 30 new genes that may cause Charcot-Marie-Tooth Disease (CMT). These genes were uncovered using computer analyses of biological data, a process called bioinformatics. This study was published…
Tarsius Pharmaceuticals has just announced positive results for their recent trial for uveitic glaucoma. This trial was called GADOT 20/20 and it was investigating a therapy called TRSo1. Ocular Diseases…
According to a story from GlobeNewswire, the biopharmaceutical company X4 Pharmaceuticals, Inc. has just announced that the US Food and Drug Administration (FDA) has granted its investigational product candidate mavorixafor…
According to a story from Pharma Advancement, Bristol Myers Squibb recently announced encouraging findings from a phase 3 clinical trial. This study was testing its drug ozanimod (marketed as Zeposia)…
According to a story from Muscular Dystrophy News Today, a gene therapy in development by the pharma company Pfizer, Inc. was recently given Fast Track designation from the US Food…
The global biopharmaceutical company Incyte recently announced the first-ever recipient of its Incyte Ingenuity Award, which will be the Massachusetts General Cancer Center (MGCC). The purpose of this award is…
According to a story from PR Newswire, the biotechnology company REGENXBIO Inc. has recently announced plans to expand its RGX-121 developmental program. RGX-121 is being developed as a potential one-time…
According to a press release from the American Kidney Fund (AKF), the organization, which is dedicated to fighting kidney disease, has partnered with the biotech company Sanofi Genzyme in a…
According to a story from PR Newswire, the drug company CiVi Biopharma, Inc. today announced that its subsidiary Eicos Sciences, Inc. has resumed recruiting patients for its phase 3 clinical…
The US Food and Drug Administration (FDA) has recently announced that it has approved a new combination therapy for the treatment of mesothelioma, a rare type of cancer. The combination…
HCU Network America is a nonprofit organization dedicated to supporting patients with homocystinuria (HCU) and finding a cure. This October, the group is organizing HCU Awareness Month. With the goal…
The company Lidl GB has just announced a recall for Cologran Stevia tablets. These sweeteners contain aspartame, or E951. Although not harmful for most people, it is essential for this…
According to a story from PR Newswire, Chiesi Global Rare Diseases and the biopharmaceutical company Protalix BioTherapeutics, Inc. have recently announced that they have launched an Expanded Access Program (EAP)…
According to a story from BioSpace, the biopharmaceutical company Mereo BioPharma Group recently announced that its investigational therapy setrusumab has earned Rare Pediatric Disease designation from the US Food and…
Since 2012, the nonprofit organization Dysautonomia International has organized Dysautonomia Awareness Month. With the goal of spreading awareness of the condition in the medical community and among the general population,…
According to a story from the AKU Society, the European Medicines Agency (EMA) has recently announced a positive opinion on extending the label indication for nitisinone (marketed as Orfadin®). The…
Recently, the Janssen Pharmaceutical Companies of Johnson & Johnson reported that they submitted a New Drug Application (NDA) for intravenously administered UPTRAVI (selexipag). The therapy is designed to treat…
According to an article in Diagnostic Imaging, MRIs and PET scans show that Parkinson’s disease may start as either body-first or brain-first, in effect behaving like two different diseases.…
Researchers have announced their identification of a specific gene mutation that can lead to atypical hemolytic uremic syndrome (aHUS). This gene is called DGKE and it is not a part of…
A research team at Ghent University in collaboration with Imec recently released preliminary data on their artificial iris. They are calling it a "smart contact lens." By embedding microelectronics into…
Misophonia is a rare condition that causes patients to have a severe and involuntary reaction to specific sounds. The cause of the condition is still unknown. A challenge misophonia patients face…
Immune-One Therapeutics has just announced that they have dosed their first acute myeloid leukemia (AML) patient in their Phase 1 trial investigating IO-202. This therapy is an immune inhibitory receptor that works…
Recently, Blueprint Medicines Corporation ("Blueprint") shared that the European Commission (EC) approved marketing authorization of AYVAKYT (avapritinib). The therapy is designed to treat adult patients with PDGFRA D842V mutated gastrointestinal stromal…
Last week, biotechnology company Anokion SA ("Anokion") announced the FDA's acceptance of an Investigational New Drug (IND) application. Anokion submitted the IND for ANK-700, a therapy for patients with…
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