Understanding which patients are at a higher risk of poorer outcomes is essential to provide them with the best treatment. That's why so many researchers focus on signs or markers…
PNH Paroxysmal nocturnal hemoglobinuria (PNH) is a rare disease where blood cells break apart and cause the contents to circulate freely within the bloodstream. This condition is caused by a…
Many father-son duos are close, but this pairing - reported on in an article from CBS58 - shares a special bond that was strengthen by the shared experience of a…
Colette Romero's son is diagnosed with eosinophilic esophagitis (EoE), a rare disease. She discusses how, among the challenges that come with this condition, education has presented extra difficulties. Education and…
Lysogene has just announced a new study has been published in the journal EMBO Molecular Medicine which demonstrates that DGKk is able to produce long-term rescue of fragile X syndrome (FXS).…
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…
Only a few hundred cases of alveolar capillary dysplasia with misalignment of pulmonary veins (ACDMPV) have ever been identified or recorded. While there could, arguably, be more cases – especially…
Many drugs, whether they are indicated for common or rare diseases, come at very high prices. Zynteglo, an investigational gene therapy for beta thalassemia, is most likely to stick to…
According to a recent article, a new study shows that granulomatosis with polyangiitis (GPA) and tuberculosis might have shared features, making diagnosis more difficult. Granulomatosis with Polyangiitis (GPA) Granulomatosis with…
The CDC has published a warning concerning a rare disease called meningococcal disease spreading in Florida. So far in 2022, the number of documented cases has surpassed the five year…
Earlier this year, the MHRA in the United Kingdom expanded the license for Kaftrio, a treatment option for patients with cystic fibrosis (CF). When this expansion occurred, allowing for the…
According to a recent article, at the American Academy of Neurology annual meeting in 2022, the importance of disease-modifying therapies for patients diagnosed with rare pediatric disorders was highlighted. Lisa…
There are numerous ways to find treatments for rare diseases, with the major method that springs into everyone's minds being clinical trials starting from scratch. However, in many cases, drugs…
Fibromyalgia is a condition that is characterized by fatigue, memory issues, and musculoskeletal pain. It is also associated with depression, anxiety, and irritable bowel syndrome (IBS). Symptoms of fibromyalgia include…
Understanding rare diseases is integral in treating them. In order to do so, research into the underlying causes and factors behind these conditions is needed. A recent study published in…
Recently, an article highlighted one man’s life journey after being diagnosed with rare cervical dystonia. Dystonia Dystonia is a chronic movement disorder, existing in the same family as the more…
Unfortunately, there are a number of racial and ethnic disparities in healthcare which position certain groups for poorer outcomes. For example, Black individuals often have lesser access to adequate medical…
According to a recent article, a child with biliary atresia was able to celebrate her first birthday following her liver transplant. Biliary Atresia Biliary atresia is a condition occurring in…
According to a recent article, a new study is demonstrating how the effects of distal renal tubular acidosis impact a patient’s quality of life. Distal Renal Tubular Acidosis (dRTA) Distal…
Theravance Biopharma Inc. has just released results from their 2nd Phase 3 investigation of Ampreloxetine as a therapy for symptomatic neurogenic orthostatic hypotension (nOH). Symptomatic nOH NOH is a rare…
Dr. Christopher Austin, a National Institute of Health Director for Transitional Sciences, spoke at last year’s virtual conference honoring Rare Disease Day. According to an article in the NIH Record, Dr.…
Many of us are familiar with the famous story written by Roald Dahl titled Charlie and the Chocolate Factory; we know the tale of the golden ticket and Charlie's journey to…
When Isabel Blevins was just 16 years old, she caught the flu. While she recovered well at the time, her health seemingly went downhill after this incident. Suddenly, Isabel was…
Each year, there are an estimated 220,000 new cases of cryptococcal meningitis. Unfortunately, researchers hypothesize that this results in over 100,000 to 180,000 deaths; many of these are in those…
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…
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