Disruption in clinical trial procedures can be added to the list of changes that have occurred since the start of the pandemic in 2020. The clinical trial sites had always…
According to a recent article at Targeted Oncology, the FDA has approved abatacept (Orencia) under priority review for preventing moderate to severe acute graft versus host disease (GvHD). The patients…
Endometrial cancer is the most common form of cancer that develops in the uterus, and there is currently a need for more treatment options, especially when it comes to the…
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…
Homologous Recombination (HR) gene mutations play a role in genomic stability and plasticity, and sometimes work to repair cellular DNA damage. These mutations cause genetic information to be shared between…
According to a recent article in Science Daily, the WHO lists Alzheimer’s disease as the cause of dementia in approximately seventy percent of cases worldwide or a total of twenty-four…
An article recently appeared in the AAMC News celebrating the creation of The Children’s National Rare Disease Institute (CNRDI). The article described the long road patients with rare diseases must…
CAR T-cell therapy has been used as a treatment for a variety of rare diseases including relapsed or refractory diffuse large B-cell lymphoma (DLBCL), non-Hodgkin lymphoma (NHL), as well as…
According to YAHOO News, twenty-two-month-old Devdan has a rare disease called type 2 spinal muscular atrophy (SMA2) which affects nerve cells that control muscles. If left untreated progressive muscle weakness…
Results from a recent Phase 3 trial for diffuse large B-cell lymphoma (DLBCL) have just been released! This trial was called POLARIX and the results demonstrated that polatuzumab vedotin-based treatment…
A recent Phase 3 clinical trial published in Lancet Oncology has identified that camrelizumab used in combination with gemcitabine and cisplatin (chemotherapy) improves progression-free survival (PFS) for those diagnosed with recurrent or…
As parents, we know our son, now fourteen, the best. And my husband and I had expected that at school, they could get to know him well too. We hoped…
Have you ever heard of a “chaperone” protein? Well, the term pretty much refers to proteins which help direct the way that proteins fold, or a protein which leads “quality…
Findings from a Phase 2 clinical trial for angiosarcoma have just been announced. These findings have demonstrated that the combination of nivolumab and ipilimumab may improve outcomes for patients. This…
For patients with biliary atresia, there are limited treatment options available. Currently, the rare liver and bile duct condition may progress to end-stage liver fibrosis and cannot be prevented or…
By the time glaucoma, a group of conditions characterized by progressive optic nerve damage, manifests in symptoms, it is often too late to restore or recover lost vision. By 2040,…
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…
Gilles Salles works at the Memorial Sloan Kettering Cancer Center as the Lymphoma Service Chief. She recently sat down to discuss the necessity of new treatments for diffuse large B-Cell…
According to an August 17, 2021 news release from pharmaceutical company Marinus Pharmaceuticals, Inc. (“Marinus”), data is available from a Phase 2 clinical trial evaluating oral ganaxolone for patients…
The coronal suture is located at the top of the head, and it is responsible for separating the two parietal bones within the frontal bone of the skull. It is…
In mid-August 2021, biotechnology and pharmaceutical development company Biosight Ltd. shared that it had begun a Phase 2 clinical trial regarding one of its proprietary treatments - and that the…
Tracey Shephard and her partner David Luffingham lost their daughter to meningitis and are now sharing their story as a warning to parents and medical professionals throughout England. The 11-year-old,…
When it comes to the diagnostic process, many refer to their journey as a "diagnostic odyssey." For those with rare or underserved conditions, diagnosis often requires months - or even…
A recent study investigated the lingering effects of sleep deprivation in healthy adults. This study was conducted by Jeremy Ochab from Poland, alongside his colleagues. Sleep Deprivation Sleep deprivation or…
The FDA has just announced their approval of zanubrutinib (Brukinsa) as a therapeutic option for Waldenström's macroglobulinemia. This approval was given based on the ASPEN Phase 3 clinical trial, and…
Sign Up With a Patient Worthy Account and Share Your Rare Story
- OR -
Make a difference, share your experiences and get paid. Opt-in and join Patient Worthy's panel for paid opportunities such as surveys, market research, patient advisory panels and more.
- OR -
Make a difference, share your experiences and get paid. Opt-in and join Patient Worthy's panel for paid opportunities such as surveys, market research, patient advisory panels and more.
