When Crystal York learned that her daughter Harmony had a rare genetic disease called DHX30, she was shocked. Even more shocking is that Harmony is just one of 40…
My name is Nicholas Alves, and I am a 26-year-old male from Massachusetts. I have a completely life destroying condition called PSSD, or post SSRI sexual dysfunction. PSSD can arise…
Sounds like ‘food for thought’. The term ‘sandwich generation’ describes family members who are usually between the ages of 45 and 55 and are responsible for raising their children…
Authored by Dr. Harsha Rajasimha, Founder and Executive Chairman, IndoUSrare. Co-Authored by Dr. Padma Rammoorthy, Medical Consultant, IndoUSrare. The diagnostic journey for patients with rare diseases often becomes a prolonged odyssey,…
The number of novel drugs approved by the FDA has doubled in the ten-year period from 2012 through 2020. Investment in investigational drugs for rare diseases appears to be continuing…
Written by Jennifer Sills, Founder, CSNK2A1 Foundation If you know me, you know I have had an unconventional life, to put it mildly. More than anyone, I know that life…
According to an article in MedicalXpress News Today, current research indicates that rare diseases in the United States affect 25-30 million people. The numbers keep rising and rival diseases…
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…
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…
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