The Griffins of Harrisburg, North Carolina enrolled their son in the AGAMREE clinical trial in 2016. Their son was the first participant in the trial studying the investigational drug vamorolone…
Rare Community Profiles is a Patient Worthy article series of long-form interviews featuring various stakeholders in the rare disease community, such as patients, their families, advocates, scientists, and more.…
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…
For over 20 years, CureDuchenne, a Patient Worthy partner and global nonprofit, has moved steadfastly forward with a mission to find, and fund, a cure for individuals with Duchenne muscular…
Sarepta Therapeutics’ treatment Elevidys (delandistrogene moxeparvovec-rokl) received conditional approval from the FDA in June 2023. The designation implies that if the drug is used in accordance with its label, it…
Editor's Note: Chronic conditions and rare diseases don't discriminate, Patient Worthy and its partners are interested in amplifying the voices of those from all identities and backgrounds. If you have…
Researchers have made an important discovery which indicates that individuals are increasingly likely to contact Clostridioides difficile (C. diff) when occupying a hospital room or bed previously used by a…
The American Society of Clinical Oncology (ASCO) held its ASCO Gastrointestinal Cancers Symposium from January 18-20, 2024. During the Symposium, doctors, researchers, scientists, and other stakeholders discussed innovative science and…
CureDuchenne, a global leader in research and patient care for Duchenne muscular dystrophy (DMD), has been working to find and fund a cure for DMD for more than two decades.…
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…
Controversial Alzheimer's drug comes off the market (axios.com)
Written by Sierra Domb What Is Visual Snow Syndrome (VSS)? Visual snow syndrome (VSS) is a neurological disorder that can impact an individual’s vision, hearing, cognition, sensory processing, and quality…
According to an article published in Healthcare Dive, a recent study published in the academic journal JAMA indicated that patients receiving treatment in hospitals owned by private equity firms were at an…
Editor's Note: Chronic conditions and rare diseases don't discriminate, Patient Worthy and its partners are interested in amplifying the voices of those from all identities and backgrounds. If you have…
For the first six months of his life, Miller Gamberi seemed to be a happy, healthy boy. But his health suddenly shifted; Miller stopped wanting to eat and drink. His…
Type 1 diabetes (T1D), rheumatoid arthritis, lupus, and ulcerative colitis are all examples of autoimmune diseases. An autoimmune disease is a condition that occurs when the immune system mistakenly attacks…
Written by Dr. Donna Nicholson Continued from Part One Content Warning: Suicide, Self-harm In the predawn hours of November 9, 1999, my husband traveled east from Arizona to New…
Rare Community Profiles is a Patient Worthy article series of long-form interviews featuring various stakeholders in the rare disease community, such as patients, their families, advocates, scientists, and more.…
The immune system is constantly on the alert for cells that have sustained DNA damage. When a cell divides its DNA molecules are, to varying degrees, usually damaged. Yet researchers…
Ring chromosome 14 syndrome is an extremely rare chromosomal disorder that was first described in medical literature in 1971. An estimated 40-80 cases have since been discovered and described. So…
The agency in Guangzhou, China, that arranged three-year-old Shelby’s adoption by a family in the US disclosed to Michelle and Adam Campbell that Shelby had beta thalassemia disorder. Yet, that…
Eliza is now 13 years old and was diagnosed at the age of three with a neurodegenerative disorder known as Sanfilippo syndrome. Eliza’s mother, Dr. Cara O’Neill, a pediatrician and…
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…
An estimated one in every four women and one in every nine men is a victim of domestic violence, writes Huecker, King, Jordan, and Smock (2023). This accounts for about…
Genes serve as the intricate orchestrators of our health. By better understanding our genetic makeup, we gain invaluable insights into how our genes shape and affect us. Yet the genetic…
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