Did you know that July 23 is World Castleman Disease Day? Each year, this day is dedicated to bringing awareness to those who are affected by Castleman disease, including patients, family…
In a recent article at Porphyria News, Claire Richmond shares how writing has helped her process her diagnosis of porphyria and to feel connected to the porphyria community. Claire Richmond’s…
In the United States, the Orphan Drug Act was established to incentivize drug developers and other stakeholders to begin developing therapies for rare diseases. Orphan Drug designation is now granted…
Tuberous sclerosis complex (TSC) is classified as a rare genetic disorder that causes typically benign tumors in the brain, skin, heart, kidneys, or lungs. As described in a recent article…
Rituximab has been used to treat patients with cancers such as non-Hodgkin’s lymphoma (NHL), and various autoimmune diseases. Recently, a study sought to understand whether rituximab treatment would be safe…
According to a press release by Merck & Co. issued on June 24, the European Commission has approved the use of adjuvant Pembrolizumab to treat resected stage IIB/C melanoma. "Adjuvant"…
Sometimes, certain health conditions can increase the risk of developing other conditions. According to a new research study, this may be the case with porphyria and hepatocellular carcinoma. An article…
At the end of June 2022, biopharmaceutical company InflaRx N.V. (“InflaRx”) shared via press release that its product vilobelimab recently earned Orphan Drug designation in both the United States…
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…
On July 7, 2022, biotechnology company Akari Therapeutics, Plc ("Akari") shared via news release that the first patient in its clinical trial has completed their course of treatment. Within…
Don't forget to check out Parts 1 and 2 of our interview, where we discussed APFED, eosinophilic esophagitis (EOE), and Lori's diagnostic process. Today, in the final portion of the interview, we're…
Did you know that July is widely considered to be Sarcoma Awareness Month? During this month, many stakeholders - from patients to researchers - aim to raise awareness of sarcomas,…
A study of hereditary angioedema (HAE) patients in Brazil was featured in an article in the Angioedema News. Researchers found that patients waited on average seventeen years for a…
According to a recent article, a study has shown that keratoconus patients who do not get their timely corneal crosslinking will have permanent changes in their corneal structure. Keratoconus…
According to Cure Today, the U.S. FDA granted Orphan Drug designation to MB-106, a novel CAR-T cell therapy designed to treat Waldenström macroglobulinemia (WM). In the United States, Orphan…
In a news release, biopharmaceutical company Fulcrum Therapeutics, Inc. ("Fulcrum") shared that the first patient was dosed in the Phase 3 REACH study. Within the study, researchers are evaluating…
If you're just joining us, don't forget to take a look at Part 1 of this interview. In Part 1, we discussed Lori and Mary Jo Strobel, what eosinophilic esophagitis (EOE) is,…
The history of mesothelioma dates back to 1767 as being the earliest record of tumors found on the lining of the lungs. In 1909 J.G. Adami introduced the term…
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…
According to a story from Charcot-Marie-Tooth News, a recent study using a mouse model of Charcot-Marie-Tooth disease type 1A produced some interesting results. The researchers found that administration of theophylline…
Findings from the MITO23 Trial investigating the outcome of the chemotherapy drug trabectedin against physician’s choice to treat BRCA-mutated patients with ovarian cancer were presented at ASCO’s 2022 Annual…
Individuals with Duchenne muscular dystrophy have mutations in the gene that makes the protein dystrophin. This gene is the largest gene in the human body, with 79 exons. When there…
Food has the power to change lives, to spark connection and understanding, to raise cultural awareness, and to bring people together. Nobody knows this better than 48-year-old Lori, who spent…
According to a story from Globe Newswire, olipudase alfa (marketed as Xenpozyme) has been approved by the European Commission for the treatment of non-CNS related symptoms of acid sphingomyelinase deficiency…
The National Organization for Rare Disorders (NORD) recently held its Living Rare, Living Stronger Patient and Family Forum on June 26, 2022. This event took place both virtually and in-person…
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