Over 10,000 rare diseases have been identified globally, a large majority of which are genetic in origin. But despite the scope of rare diseases, treatment can still be difficult to…
In 2019, South Carolina Governor Henry McMaster signed Dylan’s Law into effect. This law was passed in the wake of Dylan Emery’s death. The young boy had Krabbe disease, a…
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…
Gene therapy has the potential to improve outcomes among individuals across various disease spectrums. In the case of clinical-stage biotechnology company Rocket Pharmaceuticals, the company is working to develop…
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…
In 2019, Adam Sorgi began teaching psychology, health sciences, and AP psychology at Capistrano Valley High School: the same school that he had attended as a teenager. That same…
June 17th is International CDKL5 Day. During the month of June let’s tag every day with a reminder to those in the CDKL5 community to promote collaboration and fundraising.…
Written by Kathleen Leatherbury Hi, I'd like to share my story. In hopes of 2 things. To find help and to hopefully help others. I apologize in advance, as this…
Right now, there are insufficient treatment options for familial chylomicronemia syndrome (FCS). Unfortunately, this means that individuals living with this rare genetic disease are left without much assistance. The…
Mesothelioma can sometimes be difficult to treat. The cancer can spread to nearby tissue and nerves, and often isn’t diagnosed until later stages. Admittedly, there are a number 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…
When Taya Crowell first began feeling fatigued, they didn’t feel too worried. After all, Crowell had just changed schools and was grappling with a suddenly heavier course load. But by…
Have you ever had an itch that you just can't seem to scratch? Intensely itchy skin, also known as pruritus, is a hallmark characteristic of many conditions that affect the…
Written by Lisa Matthews I know I am not alone in my suffering…Recently, I read an article published in 2018 by The National Library of Medicine where it was estimated that,…
Effective June 1, 2023, the FDA lifted its clinical hold on Foghorn Therapeutics’ Phase 1 study of FHD-286 dose escalation in patients with acute myeloid leukemia and myelodysplastic syndromes. 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…
An estimated 2-4% of people worldwide have fibromyalgia, a chronic disorder characterized by musculoskeletal pain, fatigue, and issues with mood, sleep, and memory. This disorder, which affects women more…
Past studies have shown that our gut microbiota, or the collection of microorganisms (bacteria, archaea, fungi, yeast, viruses) within our gut, plays an immense role in our health. Our…
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…
Sometimes it can be difficult to talk about our health. For people living with genital cancers, there are sometimes additional burdens: a fear of discussing a "taboo" topic, concerns…
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…
Written by Lacey Woods From a young age, I knew I wanted to be a mother, to have biological children and also to adopt. This desire stayed with me throughout…
About 150,000 people with glaucoma across the United States wear contact lenses—and they may soon have access to an even more effective wearable treatment option. According to Medical Device Network,…
An observational study of 599 survivors of COVID-19 was conducted at the Papa Giovanni Hospital in Bergamo, Italy three months after hospital admission. The patients were evaluated for thyroglobulin…
In early 2022, the U.S. Food and Drug Administration (FDA) approved Carvykti (ciltacabtagene autoleucel) for the treatment of patients with multiple myeloma. Carvykti is a BCMA-directed T cell immunotherapy.…
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