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…
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…
According to a story from GlobeNewswire, the gene therapy company Axovant Gene Therapies Ltd. recently announced that its gene therapy AXO-AAV-GM2 has been given Rare Pediatric Disease designation by the…
Patient Worthy has recently begun a partnership with the Glanzmann's Research Foundation, a nonprofit patient organization dedicated to spreading awareness about Glanzmann's thrombasthenia and finding a cure. We spoke with…
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)…
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…
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…
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…
According to a story from Targeted Oncology, an experimental combination therapy tested on patients with advanced clear cell renal cell carcinoma (ccRCC) appeared to show potential for efficacy in a…
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…
A research team from IPM.ai, a subsidiary of Swoop, and Insmed, a biotechnology company, recently collaborated on a case study that demonstrated how AI and machine learning could be used…
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…
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…
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…
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…
According to a story from GlobeNewswire, Exagen, Inc. recently launched a testing panel called AVISE® Vasculitis AAV, which is designed to help physicians get fast and accurate data for patients…
According to a story from Charcot-Marie-Tooth News, an experimental HDAC6 inhibitor called CKD-504 appeared to restore myelination in mouse and cell models of Charcot-Marie-Tooth disease type 1A in a recent…
Sign Up With a Patient Worthy Account and Share Your Rare Story
- OR -
Make a difference, share your experiences and get paid. Opt-in and join Patient Worthy's panel for paid opportunities such as surveys, market research, patient advisory panels and more.
- OR -
Make a difference, share your experiences and get paid. Opt-in and join Patient Worthy's panel for paid opportunities such as surveys, market research, patient advisory panels and more.
