As reported in I News; scientists may have found a cure for a devastating and fatal brain disease, and the world of rare brain diseases may never be the same.…
Have you ever heard of newborn screening? Basically, newborn screening is a public health service which can identify potential hormone-related, genetic, or metabolic conditions. Since early identification is so crucial…
Claire Richmond can be considered a very experienced patient. It took Claire nineteen years to receive a diagnosis for her rare disease. During that time, she had been dismissed…
Do you love golfing? Interested in making a difference for patients with amyotrophic lateral sclerosis (ALS)? If you're located in or around Oceanside, CA, then I have an event…
The FDA has a series of designations it grants to drugs or biologics intending to treat rare, serious, or life-threatening conditions. One of these is Breakthrough Therapy designation. According to…
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…
Happy Friday! This week, author Tom Seaman asks: When it comes to anxiety, are you turning your Gizmo into a Gremlin? Also, in our new Compassion Corner series, we examine…
Unfortunately, clinical trials do not always go the way that researchers intend. Sometimes, issues arise during early or preclinical studies which cause enough concern to stop or pause the…
According to MPR, the FDA recently granted Orphan Drug designation to CPI-613 (devimistat), an investigational treatment designed for patients with biliary cancer. Altogether, "biliary cancer" can consist of bile…
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