On November 15, 2022, the Rare Disease Legislative Advocates (RDLA) hosted their monthly webinar. These webinar help provide updates to the rare disease community on legislation and other policy initiatives…
Before you read on, make sure to check out Part 1 of our interview. In Part 1, Chris Peetz discusses his background, why he joined Mirum, and the process of evaluating LIVMARLI…
Before you read on, make sure to check out Part 1 of our interview, where Annie, Peter, and Patterson discuss what recessive dystrophic epidermolysis bullosa (RDEB) is, Patterson's diagnostic journey, and how…
In the United States, the FDA grants Orphan Drug designation to drugs or biologics intended to treat, diagnose, or prevent rare diseases or conditions. A rare disease is one…
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…
Reports by experts find approximately five to ten percent of cancers may be traced to hereditary genetic changes (germline mutations) which exist in each and every cell. Instead, research in…
The American Association for the Study of Liver Disease (AASLD) held its annual Liver Meeting from November 4-7, 2022 in Washington, D.C. During the meeting, Mirum Pharmaceuticals shared two late-breaker…
Annie and Peter McKenzie describe their seven-year-old son Patterson as having a “heart of gold.” Patterson is bright, funny, happy, and empathetic. Despite his insistence that he is not a…
Have researchers from Rice University and Baylor College of Medicine discovered a potential treatment option for mesothelioma? According to a news release from the U.S. National Science Foundation, who…
Before you read further, make sure to check out Part 1 of our interview, where Brittany discusses what non-small cell lung cancer (NSCLC) is, her diagnostic journey, and how she learned that…
Since her son Tobias was born, Sophia Rincon has had to embrace her new role as medical mom. According to an article in KENS5, Tobias was born with congenital diaphragmatic…
Lyme disease is often either misdiagnosed or completely undiagnosed. According to Inside Precision Medicine's report, researchers at Mount Sinai’s Icahn School recently teamed with Johns Hopkins University to improve diagnostics…
Researchers have been evaluating '1104, an investigational immune-resetting molecule, for patients with eosinophilic esophagitis (EoE). Through immune resetting, some believe that '1104 could reduce the inflammatory cascade related to…
On the one-year anniversary of her RET+ non-small cell lung cancer (NSCLC) diagnosis, 37-year-old Brittany Hawkins had spent some time reflecting on her journey. When she was just seventeen years…
According to an October 31, 2022 press release from muscle disease company Dyne Therapeutics, Inc. ("Dyne"), the company's therapeutic candidate DYNE-251 earned Fast Track designation from the FDA for Duchenne…
Over the past decade, there has been growing research into the effects of various chemicals, like herbicides or pesticides, on human health. Researchers want to understand how herbicide or pesticide…
An estimated 4 million adults within the U.S. have fibromyalgia, a chronic disorder characterized by fatigue, problems with sleep and memory, and musculoskeletal problems. Prior studies have found that a…
Over the years, there has been more research into the impact of diet and nutrition on our overall health. How does what we eat – and how it was prepared…
According to a recent article in Medical Life Sciences, the radiation dose rates used by the new FLASH radiotherapy are over three hundred times more intense than conventional treatments. The…
Is there any relationship between achalasia and esophageal cancer? According to Healio, a research study found that there does seem to be some level of correlation. In Abstract S388, presented…
From October 7-11, 2022, the AAPNational Conference & Exhibition offered insight into clinical practices, trends, and other important information within the pediatrics specialty. According to Contemporary Pediatrics, the Family Heart…
The current standard-of-care for Fabry disease is called enzyme replacement therapy (ERT). According to Fabry Disease News, researchers have been working to evaluate an experimental ERT for patients called ISU303.…
Familial hypercholesterolemia (FH) is an inherited form of very high cholesterol. Early treatment is needed for those living with FH to lower cholesterol levels and reduce the risk of heart…
Scientists at California’s La Jolla Institute are conducting a study that is the forerunner of a clinical that will be investigating human antibodies. The new vaccine will be pitted against…
Researchers and scientists have stated that it can be difficult to diagnose and treat patients with mucinous ovarian cancer. In fact, because the cancer is often not found until it…
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