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…
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…
Both Dravet syndrome and Lennox-Gastaut syndrome (LGS) are characterized by frequent and often severe seizures. In many cases, those affected require multiple medications to manage their condition and prevent epileptic…
Unfortunately, the drug development process can be difficult, especially in the rare disease sphere. Many drug developers choose to focus on larger disease states, as these are typically more…
Actor Max Adler is known for a variety of roles in films and TV shows such as Glee, Saugatuck Cures, and Switched at Birth. But did you know that…
In June 2022, the FDA approved Amvuttra, a prescription medicine designed to treat polyneuropathy (nerve damage) due to hereditary transthyretin amyloidosis (hATTR amyloidosis). This approval was huge. Typically, managing…
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…
According to a story from Buzzfeed News, Comedian Sandy Honig, best known for writing and starring in Three Busy Debras, was recently diagnosed with gastroparesis, a rare disease that impacts…
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