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…
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…
As clinicians and scientists continue to delve into RNA sequencing benefits, they are learning how RNA sequencing provides insights for people with rare genetic conditions. The learning experience will provide…
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…
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…
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…
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…
The safety, pharmacokinetics (absorption), and tolerability of the investigational eIF2B activator ABBV-CLS-7262 is being evaluated in a Phase 1b clinical study to treat patients with Vanishing White Matter (VWM) Disease.…
Omisirge (omidubicel) is the first allogeneic (from a donor) product for SCT that has been the recipient of FDA approval. The FDA's nod moves Omisirge into the realm of…
The National Organization for Rare Disorders (NORD), a Patient Worthy partner, formed in 1983 to provide support and education to people and families living with rare diseases, advocate for change…
Compassion [kuhm-pash-uhn] noun A feeling of deep sympathy and sorrow for another who is stricken by misfortune, accompanied by a strong desire to alleviate the suffering. Compassion Corner is a…
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…
For many rare diseases, gene therapy, not traditional therapeutics, is perhaps the only means to significant relief and cure. However, the path to gene therapy has been strewn with obstacles…
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