In the United States, Orphan Drug designation is granted to drugs or biologics intended to treat, prevent, or diagnose rare conditions. A "rare" condition is defined as one affecting fewer…
As discussed in Part I of this article, a recent HDBUZZ publication featured a news item stating that due to several severe adverse events occurring during the initial AMT-130 trial…
September is Dystonia Awareness Month so I want to share a little about it and how it impacts those of us who live with it. I have to be honest…
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…
Written by Christina Fleagle There are millions of people around the world living with a rare disease, and for many, you may not know it by looking at them. But…
Often when a cure for a specific rare disease has yet to be developed, patients have enrolled in clinical trials with positive results. However, FDA-approved trials have developed a reputation…
For five years, 20-year-old Christopher Backlund lived with his grandfather, with whom he had a strong and meaningful bond. Unfortunately, Christopher's grandfather, Mitchell, passed away in Februay 2021. As Christopher…
Happy Thursday! This week, we have details on the beginnings of a new trial testing CRISPR gene editing for Duchenne muscular dystrophy, how parents can play a critical role in…
Victoria Acosta has always been an active child. The bubbly two-year-old, who currently lives in Tennessee, is described by her mother Andrea Rocha as constantly moving. However, it can be…
September 7 is World Duchenne Day, a day to help spread awareness about Duchenne muscular dystrophy among the general public and in the medical field. In recognition of this day,…
Richard B. Woodward, a well-known New York art critic, recently shared his diagnosis of idiopathic pulmonary fibrosis (IPF) in an article featured in Stat News. He explains that the general life…
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…
In a late July 2022 news release, immunodermatology innovator KSL Beutner Laboratories shared that it had launched an indirect immunofluorescence (IIF) serum blood test within the United States. The IIF…
The gut-brain axis has recently been mentioned in connection with various disorders. According to an article in SciTechDaily, a study at ECU’s Centre for Precision Health confirms a genetic…
The Family Heart Foundation has a Mission. There is so much more to accomplish and you can help prevent heart attacks by spreading awareness. This could save future generations by…
David Guy, parent of Kaila who was diagnosed with acid sphingomyelinase deficiency (ASMD), recounts his initial shock when he was told of Kaila's rare disease. David and his wife…
When we ‘do the math’ it adds up to the novel AMT-130 gene therapy receiving positive results from uniQure’s clinical trial investigating a small group (N=4) of individuals who were…
MSN recently carried the gripping story of how Elizabeth Finch, a Greeley Colorado police detective, spent last year fighting for her life due to complications from an autoimmune disease…
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…
Dr. Jung-Mo Ahn, associate professor at Dallas University in Texas, created a molecule that kills a broad range of treatment-resistant cancers. The newly targeted group includes triple-negative breast cancer.…
On August 25th, 2022, the American Society of Gene & Cell Therapy held a webinar as part of its Lunch and Learn series, which is intended to help people living…
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…
Within the first few months after Reuben Jayce-Mills was born, he seemed to be progressing fairly normally. But when Reuben turned nine months old, his parents noticed some concerning…
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…
Every day, Jackson Garwood’s parents, Darren and Rebecca, look at him and smile. There were points when they didn’t expect to have their son for this long. You see, Jackson…
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