When Cheryl Navar decided to have a child, in-vitro fertilization (IVF) seemed like the right move. Navar knew that she had a cancer gene and didn’t want to transfer…
In August 2021, the U.S. Food and Drug Administration (FDA) approved Opdivo (nivolumab) for the adjuvant treatment of a rare form of cancer called urothelial carcinoma. More particularly, the drug…
Proteinuria refers to excess protein in the urine. In healthy individuals, proteins pass through the kidneys and into the bloodstream. Proteinuria often indicates kidney dysfunction or damage. In fact,…
There have been rising incidences of gastric cancer on a global scale. As a result, this cancer is now considered the fourth leading oncological cause of death worldwide. Gastric cancer…
A Phase 2a study sought to compare the safety, efficacy, and tolerability of ABBV-3373, as compared to adalimumab, for patients with rheumatoid arthritis (RA). Patients involved in the study had…
Clinical trials can play a crucial role in understanding diseases, as well as identifying potential therapeutic options. This is especially important in newly identified or poorly understood conditions such as…
Over 30 million people in the European Union (EU) are living with a rare disease. Yet rare disease drug development is still somewhat stifled. Orphan designation helps to incentivize drug…
In a recent news release from biotechnology company Cartesian Therapeutics, the company shared that the first patient was dosed in a Phase 2b study evaluating Descartes-08 for generalized myasthenia…
High-risk neuroblastoma can be difficult to treat in children. Some research suggests that between 40-50% of children with high-risk neuroblastoma relapse following treatment, causing poor outcomes. When Flora Gentleman was…
In 2021, the U.S. Food and Drug Administration (FDA) approved zanubrutinib (Brukinsa) for adult patients with relapsed or refractory (R/R) marginal zone lymphoma. At the 64th Annual American Society for…
Currently, C5 inhibitors such as eculizumab and ravulizumab are considered the standard-of-care for people living with paroxysmal nocturnal hemoglobinuria (PNH). Unfortunately, not all patients respond well to these therapies.…
While alveolar soft part sarcoma (ASPS) is considered to be relatively slow-growing, it is necessary to carefully manage this rare soft tissue sarcoma to avoid metastasizing. Surgery is one…
.jpg)
Growing up, Priscilla Veneklause watched as her father struggled to manage his rare genetic disorder: adrenoleukodystrophy (ALD). Unfortunately, her father lost his fight with ALD when Priscilla was still…
In a mid-November 2022 news release from biopharmaceutical company Apexigen, Inc., the company shared that positive interim results were available from a Phase 2 study evaluating sotigalimab, in conjunction…
The American Society of Nephrology held its annual Kidney Week from November 3-6, 2022. During this week, multiple stakeholders within the nephrology sphere met to discuss trends, clinical care…
Clinical trials are a crucial part of learning more about certain diseases, as well as better understanding the impact of various therapeutic options. For example, a clinical trial can…
Those living with diseases like Pompe disease and McArdle disease have genetic mutations which prevent their bodies from adequately breaking down glycogen, a type of complex sugar found at…
Are there therapeutic options for patients living with unresectable (inoperable) pancreatic cancer? According to an article in MedScape, researchers sought to understand whether sunitinib or a combination of peptide receptor…
Cushing’s disease (or Cushing disease) occurs when a benign pituitary growth or tumor leads to the overproduction of adrenocorticotropic hormone (ACTH). As ACTH is overproduced, the body also begins…
Currently, the standard-of-care for individuals living with hemophilia B is intravenous factor IX infusions (prophylaxis). These infusions can be time-consuming, require adherence to a strict schedule, and may not…
So far, the Phase 2 EMPOWER-CSCC-1 clinical study has evaluated cemiplimab (also known as Libtayo) in individuals with locally advanced or metastatic cutaneous squamous cell carcinoma (cSCC). Data from…
According to a late October 2022 news release from pharmaceutical company KalVista Pharmaceuticals, Inc. (“KalVista”), the company released positive data from a Phase 1 study evaluating oral sebetralstat for…
Both Libtayo (cemiplimab) and Keytruda (pembrolizumab) have been shown to be beneficial treatment options for patients with locally advanced or metastatic cutaneous squamous cell carcinoma (cSCC). But could Opdivo (nivolumab)…
Currently, there are a number of therapies which can be used to treat individuals with dermatomyositis: corticosteroids, immunosuppressive agents, intravenous immune globulin (IVIG). Typically, corticosteroids are considered a first-line treatment,…
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…
© Copyright 2024 Patient Worthy
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.
