According to a story from European Pharmaceutical Review, the European Commission has recently approved a label extension of ORKAMBI (lumacaftor/ivacaftor), a drug used to treat cystic fibrosis. Under the extension,…
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 news-medical.net, a recent study found that elevated body-mass index (BMI) was associated with worse outcomes for people living with acute lymphoblastic leukemia, with this study…
Welcome to Study of the Week from Patient Worthy. In this segment, we select a study we posted about from the previous week that we think is of particular interest…
According to an a press release published on Business Wire, Arrowhead Pharmaceuticals Inc. recently presented updated phase 2 data at the 2023 European Association for the Study of the Liver…
Patient Worthy partner TREND Community recently presented an abstract at the National Scleroderma Foundation Conference, which took place on July 14 in Orlando, FL. The abstract, presented as a poster…
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 Healio, a phase 3 clinical trial evaluating pembrolizumab plus perioperative chemotherapy as a treatment for locally advanced and resectable gastric adenocarcinoma and gastroesophageal junction cancer…
According to a story from International Business Times, a French couple encountered an unpleasant skin condition as a result of a pest infestation in their home. Their case was reported…
Welcome to Study of the Week from Patient Worthy. In this segment, we select a study we posted about from the previous week that we think is of particular interest…
In a story from BioPharma Dive, a number of high-profile clinical trials are under way that could see conclusive results by the end of 2023. While some of these are…
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…
On June 21, 2023, the Rare Disease Legislative Advocates (RDLA) hosted its monthly webinar. These webinars help provide updates to the rare disease community on legislation and other policy initiatives…
Welcome to Study of the Week from Patient Worthy. In this segment, we select a study we posted about from the previous week that we think is of particular interest…
According to a story from Healio, in a phase 3 clinical trial, treatment with the drug resmetirom was able to achieve the desired endpoints at 52 weeks in people living…
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 Healio, a speaker at the Heart in Diabetes CME Conference claimed that postural orthostatic tachycardia syndrome (POTS) is becoming a common complication for long COVID-19…
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 statnews.com, a new documentary film from Elle Brooks-Tao titled "I'm Not Crazy, I'm Sick" delves into the stories of three people living with long-term Lyme…
According to a story from BioPharma Dive, Apellis Pharmaceuticals will now join the ranks of pharma companies that have seen an investigational therapy being developed for amyotrophic lateral sclerosis falter…
Welcome to Study of the Week from Patient Worthy. In this segment, we select a study we posted about from the previous week that we think is of particular interest…
According to a story from healio.com, an oral medication being evaluated in a phase 2 study was able to increase annualized height velocity in children living with growth hormone deficiency,…
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, the QurAlis Corporation, a clinical stage biotech company, recently announced the completion of the review of the Clinical Trial Regulation (CTR) in the…
The World Orphan Drug Congress USA 2023 was held from May 23-25, 2023, at the Gaylord National Resort & Convention Center in National Harbor, MD. This conference is focused solely…
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