According to a story from Business Weekly, the biotechnology company Healx, from Cambridge, UK, and the US-based Mission: Cure coalition have announced a new partnership which aims to help develop…
According to a press release from the rare liver disease company Albireo Pharma, Inc., the US Food and Drug Administration (FDA) has accepted its New Drug Application (NDA) for odevixibat.…
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…
Patient Worthy is excited to announce that this Saturday a solidarity concert will be held in Noáin in favor of Dravet syndrome. United by the Dravet through Ranchera Music…
According to a story from Yahoo! Finance, EUSA Pharma Limited and BeiGene, Ltd. have recently announced that their Biologics License Application (BLA) has been accepted by the China National Medical…
On January 21, 2021, the Rare Disease Legislative Advocates (RDLA) hosted an online webinar that focused specifically on the subject of advocating for rare disease health policy at the state…
Welcome to study of the week, a new series from Patient Worthy. In this segment, we select a study we posted about from the previous week that we think is…
According to a story from newswise.com, a team of researchers from the University of New Hampshire may have discovered the next new treatment for a rare form of blood cancer…
Written by Gina Glass Like any parent of a child with a rare disease, my world was turned upside down when my daughter, Gia, was diagnosed with sickle cell disease…
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 study on hindawi.com, the presence of abnormally elevated levels of serum IgG4 was correlated with a greater likelihood of patients living with autoimmune hepatitis to present with…
According to a story from GlobeNewswire, a new partnership between the precision genetic medicine company Akouos, Inc. and and Blueprint Genetics has produced the Resonate program, which aims to improve…
According to a story from news-medical.net, a team of scientists affiliated with Massachusetts General Hospital has conducted a study revealing that gene therapy could be an effective treatment for tuberous…
According to a story from Medscape, in a recent study the drug capivasertib appeared to be effective as a treatment for certain metastatic cancer tumors. Classified as an inhibitor of…
According to a story from Globe Newswire, the genetic medicine company Sarepta Therapeutics has announced top-line data from part one of its clinical trial. This trial is investigating its experimental…
Veer Basal, age 14, first started showing signs of Friedreich's ataxia when he was around ten years old. The first sign that something was wrong was when Veer began to…
Welcome to study of the week, a new series from Patient Worthy. In this segment, we select a study we posted about from the previous week that we think is…
According to a story from dernieresactus.fr, the biopharmaceutical company Inventiva has recently announced plans for its upcoming phase III clinical trial. This trial will investigate its lead candidate lanifibranor as…
Note: This press release was republished with permission from the EveryLife Foundation for Rare Diseases. The Rare Disease Legislative Advocates (RDLA), a program of the EveryLife Foundation for Rare Diseases,…
Dr. Cory M. Resnick, Oral & Maxillofacial Surgeon at Boston Children’s Hospital and Assistant Professor at Harvard Medical School, describes the exciting progress he has made on the Prenatal Diagnosis of Pierre Robin Sequence. Interview by Philippe Pakter, chairman of Pierre Robin Europe.
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, a recent cell-based study has revealed that Charcot-Marie-Tooth disease type 1C and type 4J share a common pathway malfunction. This is despite 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…
Welcome to study of the week, a new series from Patient Worthy. In this segment, we select a study we posted about from the previous week that we think is…
According to a story from Targeted Oncology, Dr. Bradley Monk talked about the importance of molecular testing in determining the treatment course for ovarian cancer at the Targeted Oncology Case…
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