With the overall focus of the Reuters Pharma & Patient USA 2020 conference being the relationship between patients and drug manufacturers, a key topic focused on how companies can form…
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 BioNews, a recent study has revealed more information about the maternal protein SMCHD1, and impacts to the normal function of this protein could play a…
According to a case study published in Hindawi, a case study of a 36 year old Black woman provided an unusual and rare example of intestinal pseudo-obstruction presenting as the…
When she was born, Amy Parr was completely paralyzed from the neck down. Doctors were not optimistic about her future. Some said that she would not make it beyond her…
The Reuters Pharma & Patient USA 2020 virtual conference included a number of shorter presentations that focused on specific approaches to help drug companies better meet the needs of patients.…
According to a story from Charcot-Marie-Tooth News, the National Organization for Rare Disorders (NORD) is beginning a new initiative that will be the focus of a public virtual meeting that…
According to a story from SMA News Today, a recent case study has reported instances in two spinal muscular atrophy (SMA) patients in which repeated administrations of the therapy nusinersen…
According to a story from BBC, Xavier Alford woke up one morning with unusual tingling that affected his toes and fingers. Before long, his legs began to weaken. That was…
According to a story from GlobeNewswire, the biopharmaceutical company MediciNova, Inc. has announced encouraging results related in Optical Coherence Tomography (OCT) in a phase 2b clinical trial. This clinical trial…
According to a story from ktla.com, a man from Nevada in his twenties has died after being hospitalized. The man was killed by hantavirus and was likely exposed to the…
Author: Harsha K Rajasimha, MS, Ph.D. Editor: Sharlene Brown, Ph.D. When clinical investigators and study coordinators start planning a new study design, speed and efficiency are paramount. Running a clinical…
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 Markets Insider, the biotechnology company REGENXBIO Inc. has recently released data from its phase 1/2 clinical trial. This is interim data from cohorts 1 and…
According to a story from Globe Newswire, the biopharmaceutical company Ultragenyx Pharmaceutical Inc. has recently announced that its investigational treatment UX701 has earned the Orphan Drug designation from the US…
According to a story from Cancer Therapy Advisor, a recent phase 1 clinical trial investigated an experimental CAR T-cell therapy in heavily pretreated patients living with multiple myeloma, a rare…
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…
Ever since he was a young boy, JR Trout, who lives with spinal muscular atrophy (SMA), had a sense that there was something different about him. For years, he never…
According to a story from blogs.biomedcentral.com, a research project called SCOPE H2020 focused on the impacts of chronic kidney disease screening of the elderly in the EU. The research included…
According to a story from spectrumnews1.com, three year old Israel Bombela has endured many health challenges in his short life so far. The youngster went through a bout of COVID-19…
According to a story from Newswise, the early results of a recent phase 1/2 clinical trial have revealed the potential of an experimental treatment that was being tested for blastic…
A recent study sought to evaluate the impact of family history on the risk of autoimmune diseases, particularly primary biliary cholangitis and autoimmune hepatitis. There hasn't been much research conducted…
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…
From December 1-4, 2020, Reuters hosted a virtual conference called Pharma & Patient USA 2020. The primary focus of this event was furthering patient centricity in disease development as well…
According to a story from Hematology Advisor, a recent study released in Scientific Reports indicates that gene expression has the potential to predict both primary resistance and treatment response in patients living…
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