Popular Canadian singer Justin Bieber stunned the world when he announced he was diagnosed with Ramsay Hunt syndrome following his facial paralysis. Ramsay Hunt Syndrome Ramsay Hunt syndrome (herpes zoster…
Thanks to Amylyx Pharmaceuticals, Canada becomes the first country worldwide to have access to an ALS drug branded Albrioza (originally AMX0035). According to an article in Yahoo News, the drug…
In the United States, the FDA grants Orphan Drug designation to drugs or biologics intended to treat, prevent, or diagnose rare diseases or conditions. Do you know what makes a…
Meghan Rourke saw the need for more open conversations about chronic illnesses, sparking her to write The Invisible Kingdom: Reimaging Chronic Illness. In an excerpt from her novel, she shares…
From June 11th until the 14th, the Society of Nuclear Medicine & Molecular Imaging (SNMMI) Annual Meeting was held in Vancouver, Canada. At this meeting, Attralus Inc. shared new data…
In clinical trials evaluating Zolgensma for children with spinal muscular atrophy (SMA), the maximum weight of participants was 18.7 pounds. However, parents with children weighing over that weight might be…
In normal development, genes are usually inherited in pairs - with one copy from the father and one inherited from the mother. When one copy of the TCF4 gene, which…
At just two days old, Leita’s son Glyn started having infantile spasms. For the next sixteen years, Glyn’s seizures worsened. Leita also had two other children – twins named Asha…
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…
Jonathan Rayner works at the University of South Alabama as the Director of the Laboratory of Infectious Diseases. Jonathan has studied ticks since back in 2018. He and his colleagues…
When Alicia McLeod entered her 20s, she began noticing that she was showing a few concerning symptoms. Eventually, Alicia visited the doctors, where she was diagnosed with syringomyelia. According to…
According to a recent article, a 6(b) study of pharmacy benefit managers and anticompetitive business practices was approved by the Federal Trade Commission following pressure from the NCPA. The Study…
Global Blood Therapeutics (GBT) has just announced that their therapeutics called GBT021601 (GBT601) and inclacumab for sickle cell disease (SCD) have received both Rare Pediatric Disease Designation and Orphan Drug…
Andrew is a pretty tough kid - he had already undergone four open heart surgeries by the time he was three years old. Now, at age 21, he's added two…
June 19, 2022 will be recognized as World Sickle Cell Day. This international event is intended to help spread awareness about sickle cell disease among the general public and in…
Currently, no approved treatment options exist for children with pediatric low-grade glioma, the most common brain tumor found in children. However, shares Day One Biopharmaceuticals ("Day One") in a news…
Anemia is a common symptom for chronic kidney disease (CKD), but there hasn't been much research done on the association between the two or the adverse outcomes which ensue as…
In the past, research has shown that bleach baths can be a helpful tool in addressing or relieving symptoms associated with dermatological issues. For example, some doctors suggest the use…
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…
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…
At the end of May, 2022, the Governor of Connecticut, Ned Lamont, established a permanent Rare Disease Advisory Council (RDAC) for the state by signing House Bill 5500 (HB 5500).…
When Lauren Atkins and William Bromley welcomed their son Charlie into their lives, they were thrilled. Jessie, their daughter, may have been even more thrilled, already having a soft spot…
The FDA has just announced that they have lifted the temporary hold placed on pheNIX gene therapy. This gene therapy was created by Homology Medicine as an investigational treatment for…
The FDA has recently approved combination treatments for those diagnosed with unresectable advanced or metastatic esophageal squamous cell carcinoma. These combination treatments are nivolumab and fluoropyrimidine along with platinum-containing chemotherapy…
According to a story from news-medical.net, the International Rett Syndrome Foundation (IRSF) has announced the launch of its Clinical Trial Committee. The committee was implemented with the goal of helping…
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