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…
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…
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