According to a story from finance.yahoo.com, a new treatment regimen has been approved by the US Food and Drug Administration (FDA) for hepatocellular carcinoma that cannot be treated with surgery.…
Have you ever wondered how a rare disease can take control of a person’s life? The deteriorating health, being unable to receive an accurate diagnosis, and more importantly finding an…
Genetech recently announced the FDA’s approval of Tecentriq to treat alveolar soft part sarcoma (ASPS) pediatric and adult patients over the age of two. This first-ever treatment has been developed…
An announcement via PR NewsWire by NeuroSense Therapeutics based in Cambridge, Massachusetts carried the results of its multi-dose study of PrimeC to treat amyotrophic lateral sclerosis (ALS) Expectations are…
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…
When we look at statistics that show thirty million people in the U.S. has one of seven thousand rare diseases, they may not seem rare. However, when we realize that…
The CDC lists lung cancer as the third most common type of cancer in the United States with the highest death rate. Estimates are given of 2.2 million patients worldwide…
Medscape Medical News reports that results have been received from the OCCLURANDOM Phase 2 trial which is the first randomized trial investigating the antitumor effect of the peptide receptor radionuclide…
Diffuse midline glioma (DMG) refers to a rare and extremely aggressive brain tumor that is most common in children. Chemotherapy and radiation are the current standards-of-care. Unfortunately, even with…
Dr. Jose Sandoval-Sus, an oncologist at the Moffitt Cancer Center, was recently interviewed with Medscape regarding his review which was published in the October 2022 Current Oncology Reports. The new…
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…
At 45 years old, Tiffany Wedekind has embodied a sense of gratitude. Each day, she wakes up happy to be alive – and aims to bring that happiness to others.…
In the past, there have been a small number of studies exploring the connection between sickle cell disease (SCD) and craniosynostosis. More recently, researchers sought to understand whether there was…
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…
Research has shown that up to 70% of individuals who undergo stem cell transplantation experience organ damage within just a few months. Many develop graft-versus-host disease (GvHD), a complication in…
Week 13 of the NFL marks the onset of “My Cause My Cleats,” an annual initiative that empowers players to reveal and share their passions beyond the game. This year…
According to a story from morningstar.com, the pharmaceutical company Horizon Therapeutics plc recently announced the completion of enrollment for its phase 3 clinical trial in Japan. This trial is testing…
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…
To those within the rare disease community, it is no surprise that therapies and drugs can sometimes be exorbitantly expensive. However, Spain’s National Markets and Competition Committee (CNMC) is…
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…
According to a story from CNN, a new treatment was recently approved by the US Food and Drug Administration (FDA) for amyotrophic lateral sclerosis (ALS), also known as Lou Gehrig's…
In July 2022, Global Genes, a nonprofit 501(c)(3) organization and global advocate for those with rare diseases, awarded its Health Equity in RARE Patient Impact grants to members of…
In the United States, Orphan Drug designation is granted to drugs or biologics intended to treat, diagnose, or prevent rare diseases or conditions. These are defined as conditions affecting fewer…
Before you read on, don't forget to check out Part 1 of our interview. In Part 1, we discussed Dr. Pariser's background, what hyperhidrosis is, and its symptoms and manifestations. Today, we'll…
Inside Precision Medicine recently featured an article focusing on biomarkers that suggest the likelihood of drugs called checkpoint inhibitors to “backfire” during melanoma treatment. Checkpoint inhibitors are one of the…
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