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 Charcot-Marie-Tooth News, the drug company DTx Pharma has recently obtained critical funding that will go towards the development of new RNA-based treatments for Charcot-Marie-Tooth disease…
According to a story from BioSpace, SIRION Biotech GmbH and Mustang Bio, Inc. have announced a new agreement for licensing the use of SIRION's proprietary LentiBOOST™ technology for use by…
According to a story from BioSpace, the US Food and Drug Administration (FDA) recently announced that it has awarded grant funding that will go towards six clinical trials that will…
According to a story from Newswise, it was more than three years ago that Maxford Brown, 16, awoke one morning feeling out of sorts. Maxford, who lives with Down syndrome,…
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 BioSpace, the biopharmaceutical company Kite announced recently that the Committee for Medicinal Products for Human Use (CHMP), part of the European Medicines Agency, has released…
According to a story from Vice World News, the Solanki family, who are residents of Ahmedabad, the largest city in India's western state of Gujarat, recently took action to save…
In a recent press release the US Food and Drug Administration (FDA) has officially approved remdesivir (marketed as Veklury) as a treatment for cases of COVID-19 that require hospitalization. It…
According to a story from BioSpace, the biotechnology company Ascentage Pharma announced that two of its investigational treatments have recently received Orphan Drug designation from the US Food and Drug…
According to a story from BioSpace, a recent study conducted by the biotechnology company Goldfinch Bio in collaboration with academic partners is highlighting the importance of proteinuria in determining outcomes…
On October 15th, 2020, the Rare Disease Legislative Advocates (RDLA) held a webinar discussing some of the latest news regarding ongoing legislation that is most relevant to the rare disease…
According to a story from BioSpace, the gene therapy company Axovant Gene Therapies Ltd. recently announced that it has been given Rare Pediatric Disease designation from the US Food and…
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 medical device company SoniVie announced recently that the US Food and Drug Administration (FDA) has granted approval for its Investigational Device Exemption (IDE).…
According to a story from Market Watch, the biopharmaceutical company Alexion Pharmaceuticals, Inc. has recently announced that the US Food and Drug Administration (FDA) has approved a new formulation of…
On October 14th, 2020, the International Waldenström's Macroglobulinemia Foundation (IWMF) hosted a webinar featuring Dr. Irene Ghobrial of the Dana Farber Cancer Institute. The focus of her presentation was on…
Dr. Justin Hopkin's son Garrett was born happy and healthy, but when he was around four to six months old, his parents began to notice some problems. Garrett was having…
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…
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