A group of autoimmune diseases called anti-neutrophil cytoplasmic autoantibody (ANCA)-associated vasculitis (AAV) is the subject of a preprint under consideration at Pediatric Rheumatology. ANCA-AAV is characterized by damage to small…
Rare cancers are a unique research area which necessitate specified attention. Cancer Commons, Pattern.org, and the Rare Cancer Research Foundation are three organizations dedicated to improving the lives of those…
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As we know, it can be incredibly difficult to receive the proper diagnosis for a rare disease. In fact, it typically takes at least seven years and numerous doctor's appointments.…
In a news release from early February 2022, clinical-stage pharmaceutical development company Algernon Pharmaceuticals Inc. ("Algernon") shared that its Phase 2 clinical trial evaluating Ifenprodil (NP-120) was fully enrolled. Within…
The Marfan Foundation has a mission to improve the quality of life for patients living with Marfan syndrome and other aortic and vascular conditions. This month, in honor of Marfan Syndrome…
On February 7, 2022, Healthline reported that the FDA had approved Vabysmo (faricimab-svoa), an injectable therapy for those with wet age-relate macular degeneration (wAMD) and diabetic macular edema. This approval…
A tragedy in Deer Creek, Oklahoma has pushed bacterial meningitis into the spotlight after it claimed the life of Nathan Rogalski. Nathan, who was a star athlete within his community,…
According to a recent article, researchers have conducted a pilot human trial to test out a falloposcope which captures images of the fallopian tubes in patients, which will hopefully enable…
If you would like to read the first part of this interview, please take a look at Part 1 here. In the first part of the interview, I spoke with…
The Ebola virus is endemic in certain areas of Africa, where viral outbreaks still exist to this day. For example, there were at least three Ebola outbreaks in Africa during…
February 15, 2013 marked the first-ever International Angelman Day. The day, still celebrated nine years later, aims to raise global awareness for Angelman syndrome. Additionally, International Angelman Day serves to…
HTT gene mutations cause Huntington’s disease, a rare and inherited neurological condition. Those with a family history of Huntington’s disease may choose to undergo genetic testing to learn more, though…
LOXO-305 is an investigational Bruton’s tyrosine kinase (BTK) inhibitor which is currently in development for the treatment of mantle cell lymphoma (MCL). The next step is to evaluate LOXO-305 in…
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…
According to a recent article, a study in Estonia looked at the prevalence and characteristics of spinal muscular atrophy (SMA) over the last 24 years to aid in implementing SMA…
Have you ever heard of comorbidities? In short, comorbidities mean the presence of more than one disease or condition in someone at a time. For example, arthritis and heart disease…
Clinical trials, drug development, and medical research have the potential to change the therapeutic landscape for patients with a wide variety of conditions. At ProQR, founder and CEO Daniel de…
Betty Vertin recently wrote an article discussing her experience as a parent of a child with Duchenne muscular dystrophy. She shares the lessons she has learned in order to…
If you have chronic pain like I do (mine is caused by a movement disorder called dystonia), or one of many other thousands of health conditions, or just dealing with…
Digital health company AEYE Health was founded on the desire to provide fully automated, artificial intelligence (AI) based diagnostic screening tools for retinal imaging. According to the Ophthalmology Times, the…
Misconceptions exist about many rare diseases. In fact, the efforts of many advocates are focused on education and dispelling these myths. Recently, an article in the Hindustan Times did just…
In a news release from late January 2022, biopharmaceutical company CASI Pharmaceuticals, Inc. ("CASI") shared that the Company's partner, BioInvent International AB ("BioInvent") recently earned an Orphan Drug designation from…
Rare Disease Week 2022 is taking place this year on February 22nd to March 2nd. Though normally held in Washington, DC, the EveryLife Foundation for Rare Diseases, which organizes the…
A year ago, Paddy Doherty’s doctor told him that he had a rare hereditary disease called transthyretin (ATTR) amyloidosis, the same disease that had killed his father. As reported in…
Renal cell carcinoma (RCC) is a highly metastatic (easily spreading) cancer. If RCC progresses, it can spread to other organs, such as the lungs. Although RCC is a rare cancer,…
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