The 25th World Congress of Dermatology took place this year from July 3 to 8, 2023. During the Congress, stakeholders throughout the medical industry convened to discuss research, trends, and…
Professor Timothy Yu developed the custom drug milasen named in honor of Mila, an 8-year-old girl with Batten disease. The drug is the first drug specifically designed for one…
Currently, there are an estimated 10,000 known rare diseases (and counting). As we learn more about our genetic makeup, and how genetic alterations can cause disease, we continue to…
The Peter Pan novel which J.M. Barrie wrote in 1911, based on his 1904 play, includes a curious concept about the hideout trees Peter uses for his lost boys' housing.…
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…
American consumers have access to the most advanced pharmaceutical systems in the world. Recent medical and technical advances seem to support that reputation. According to Medical Xpress, a Swiss…
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…
Want to learn about scientific topics without needing a PhD? Check out the Science Simplified blog from TESS Research Foundation! Dr. Tanya Brown, PhD, works with researchers to make science…
Dr. William Lynes completed his residency in 1987 at Stanford University. He began his practice at the Kaiser Permanente Riverside Medical Group in California. It was the idyllic life that…
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…
In 2015, rare disease stakeholders in North Carolina established the first Rare Disease Advisory Council (RDAC); the National Organization for Rare Disorders (NORD) explains that an RDAC is an…
Tobacco consumption has been heavily linked to the development of chronic obstructive pulmonary disease (COPD). While not all smokers will develop COPD, a majority of COPD cases do result from…
Aboard the private yacht, the crew and passengers delighted in the sights of the open water. It was relaxing onboard: a welcomed break from the pressures of daily life. The…
Rheumatoid arthritis, lupus, multiple sclerosis, and myasthenia gravis are all considered autoimmune disorders. These conditions occur when the immune system, which normally protects the body against infections or other…
Want to learn about scientific topics without needing a PhD? Check out the Science Simplified blog from TESS Research Foundation! Dr. Tanya Brown, PhD, works with researchers to make science…
Uplifting Athletes, a national nonprofit organization that harnesses the power of sport to support people impacted by rare diseases, celebrated 20 years of Lift for Life with Penn State University…
A recent article published in GlobeNewswire captured the results of the trial that was presented at the 2023 Annual ASCO Meeting from June 2nd to June 6th, 2023 in Chicago.…
When it comes to the rare disease landscape, there is not enough conversation discussing mental health needs. People undoubtedly need more mental health support and resources during the diagnostic odyssey.…
I've known some wonderful fathers throughout my life. My own father was a gem. My siblings agree. However, it was not until I became immersed in the rare disease world…
Rare Community Profiles Rare Community Profiles is a new Patient Worthy article series of long-form interviews featuring various stakeholders in the rare disease community, such as patients, their…
In August 2022, I reported on a story that touched my heart: Lucy Landman's PGAP3 diagnosis. With less than 50 diagnosed cases of PGAP3 worldwide, parents Zach and Geri jumped…
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 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…
WebMD recently carried a story about a young woman who, after nine years of wheelchair confinement caused by a rare metabolic disease with no name, was able to walk…
The FDA has approved the drug Ayvakit developed by the biotechnology company Blueprint Medicines for the treatment of indolent systemic mastocytosis. According to an article in BiopharmaDive, Ayvakit had been…
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