According to a story from StokeonTrentLive, 34-year-old mother Rachel Potter is living with thousands of tumors thanks to a rare genetic disorder called neurofibromatosis type 1 (NF1). Though they aren't…
Prednisone, oral steroids, intravenous hydrocortisone, and even blood transfusions are used to treat autoimmune hemolytic anemia (AIHA). AIHA comprises of warm AIHA, mixed AIHA, or cold agglutinin disease (CAD). Some…
Written by Carol Trager Parents of a “rare” child constantly search for answers, for direction, for hope. They collect information and advice from a variety of sources, yet they must…
Patient Worthy's award-winning podcast "Wait, How Do You Spell That? A Rare Disease Podcast" is back with a new episode. This week, Colby is sitting down with Dr. Tracy Dixon-Salazar,…
A team of scientists from Japan appear to have pioneered a method for reversing synapse damage in an Alzheimer's disease mouse model. Although encouraging, the theory must be tested in…
According to a story from Scleroderma News, findings from the recent Phase 2 EDITA clinical trial suggested that ambrisentan (marketed as Letairis), which is approved to treat pulmonary arterial hypertension…
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.…
When he was just three years old, Florida State Seminoles outfielder Jaime Ferrer was diagnosed with type 1 diabetes, a chronic condition where the pancreas makes little to no insulin.…
The idiom ‘third time’s a charm’ may have some significance in this instance. The panel that advises the FDA just voted unanimously in favor of Eli Lilly’s experimental drug, donanemab.…
Cholera is an acute bacterial disease caused by Vibrio cholerae that can lead to severe diarrhea, vomiting, and even death. Although only 10% of cholera cases are considered severe, identifying…
Long COVID is a phenomenon emerging from the global spread of SARS-CoV-2, a strain of coronavirus causing COVID-19. Seven million people died from COVID-19, though this is considered a serious…
Living with a chronic health condition is sometimes reminiscent of the plight of Sisyphus, a king from Greek mythology. Sisyphus was king of Ephyra who was punished for chronic deceitfulness…
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.…
Professor Michael Goran was referring to research data that was presented at the annual 2023 conference of the Hepatology, Gastroenterology, and Nutrition Society of North America when he stressed that…
Sometimes, the itch from seborrheic dermatitis can be - frankly - unreal. This common, chronic skin condition causes flaky, greasy, scaly, and itchy skin on different areas of the body.…
Rare diseases are challenging. Not just for those affected, but for researchers, physicians, and drug developers. Rare diseases may affect small populations. Many are poorly understood and lack adequate awareness.…
According to a story published on Healio, a recent study of patients living with Crohn's disease that had resulted in a perianal fistula found that patients that maintained a concentration…
An estimated 80% or more of people with myotonic dystrophy type 1 (DM1) struggle with severe fatigue and excessive daytime sleepiness (EDS). While most people consider myotonia (inability to contract…
Researchers have previously linked TREX1 gene mutations to the development of a rare inherited small vessel disease called retinal vasculopathy with cerebral leukoencephalopathy (RVCL). What researchers didn't know was why these genetic…
Stemnovate is a biotechnology company working to transform the drug discovery landscape while improving diagnostics. Currently, the company hopes to develop a research platform for rare pediatric mitochondrial diseases such…
The month of July is recognized as Sarcoma Awareness Month, a time of the year for spreading awareness about sarcoma among the general public and in the medical community. An…
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.'…
Patient Worthy's award-winning podcast "Wait, How Do You Spell That? A Rare Disease Podcast" is back with a new episode. This week, Colby is sitting down with Brandi Benson, a…
Mirum Pharmaceuticals has long been a leader in the rare liver disease space, driving forward with a mission of empowering patients and families, and identifying novel therapeutic interventions to help…
Ruud Dobber, executive VP of biopharmaceuticals at AstraZeneca was quoted in a press release dated saying that type 2 diabetes is on the rise in adolescents and younger children with…
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