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…
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…
According to a story from Healio, the pharmaceutical company Revolo Biotherapeutics has been developing '1104, a peptide with a mechanism that 'resets' the immune system to pre-empt the appearance of…
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 recent study found that a treatment combination of adalimumab and low dose methotrexate was able to reduce treatment failure by two times in…
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…
According to a story from BioPharma Reporter, the company bluebird bio has recently submitted its Biologics License Application (BLA) to the US Food and Drug Administration (FDA). This application is…
According to a story from CBS News Boston, Scarlett is just six years old and was recently hit with a devastating diagnosis: Batten disease. Unfortunately, very little can be done…
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…
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 May 18, 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…
An announcement from the drug company Genentech highlights the results of a recent study which demonstrated that the drug faricimab-svoa (marketed at Vabysmo) was able to dry retinal fluid more…
The month of May is recognized as Prader-Willi Syndrome (PWS) Awareness Month, and May 27th is International PWS Day. This is a time to spread awareness about this rare disease…
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…
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 April 25, 2023, myTomorrows, a platform designed to help patients obtain access to experimental medicines, recently launched a new tool called TrialSearch AI, an artificial intelligence-powered resource for physicians…
Marsha Calloway-Campbell lives a full life as a lawyer, business owner, wife to her husband Armaray, mother to her three adult daughters, and “Naun” to her toddler grandson. But her…
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, a recent study found that patients with metastatic pancreatic cancer (more specifically, pancreatic ductal adenocarcinoma, the most common form) that were being treated with…
According to a story from Healio, the US Food and Drug Administration (FDA) has recently updated the indication for the drug teprotumumab (marketed as Tepezza). Approved for the treatment of…
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…
May 7, 2023 will be recognized as Cystinosis Awareness Day. This will be a day to spread awareness about the rare disease cystinosis among the general public and the medical…
According to a story from PR Newswire, the biopharmaceutical company Avidity Biosciences, Inc., recently announced that its investigational therapy AOC 1044 has received the US Food and Drug Administration's Fast…
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 Endpoints News, the drug developer Incyte has opted to put an end to its phase III clinical trial evaluating parsaclisib, an orally available PI3K inhibitor,…
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