Kaelyn Franco was an experienced athlete by the time she entered college, and a friend recommended taking a spin class. According to an article in Yahoo, 23-year-old Kaelyn took her…
According to a recent study by the investigative staff at Tufts University in Boston, Massachusetts, there is a direct correlation between the ethnicity or race of the site personnel and…
The FDA, the NIH, 5 nonprofits, and 10 pharmaceutical companies, have formed an official partnership to accelerate gene therapy development for rare diseases. Out of 7,000 known rare diseases, just…
Because it is Thanksgiving, the Patient Worthy team asked me to share my thoughts on gratitude. Some among us might think it would be a daunting assignment. After all, I have a…
As someone struggling with chronic illness, you are probably no stranger to the stomach problems that come along with treatment or just a reality of your disease. By now, you might…
The Rare Disease Diversity Coalition and Global Genes have recently established a new partnership. This partnership aims to help find solutions to help address outcome disparities faced by people of…
Compassion [kuhm-pash-uhn] noun A feeling of deep sympathy and sorrow for another who is stricken by misfortune, accompanied by a strong desire to alleviate the suffering. Compassion Corner is a…
According to a recent article, the overall survival of patients with untreated metastatic non-small cell lung cancer (NSCLC) were significantly improved by the addition of two immunotherapeutic drugs to…
Want to learn about scientific topics without needing a PhD? Check out the Science Simplified blog from TESS Research Foundation! Dr. Tanya Brown, PhD, works with researchers to make science…
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…
In a recent article by Bailey Martens, she describes the struggles she and others have faced being disabled as journalists and brings light to the importance of accessibility in the…
November is officially recognized as National Family Caregivers Month, a time dedicated to recognizing and celebrating caregivers. The entire month is full of recognizing the effort and needs of caregivers,…
The FDA grants certain designations, such as the Orphan Drug (ODD) and Fast Track designations (FTD), to medications intended to treat rare and severe disorders. Recently, they granted both of…
According to a recent article, patients diagnosed with a rare disease are faced with incredibly high health care costs compared to those without a rare disease. Impact of Rare Disease…
On October 27, 2021, the National Organization for Rare Disorders (NORD) hosted an informational webinar titled "Navigating Diversity, Equity, and Inclusion (DEI) in Rare Disease Non-Profits." This webinar was the…
Access Opportunities for Rare Disease Patients At present, there are around 7,000 identified rare diseases, with only 5% having treatments. That leaves more than 95% of rare disease patients with…
When a disease is progressive, it can be difficult to discern if treatment is working. Transthyretin amyloid cardiomyopathy (ATTR-CM) is an example, especially as its treatment, tafamidis, presents "relatively few…
Compassion [kuhm-pash-uhn] noun A feeling of deep sympathy and sorrow for another who is stricken by misfortune, accompanied by a strong desire to alleviate the suffering. Compassion Corner is a…
Aurion Biotech has just released the results from their clinical trial called IOTA. This trial was conducted by researchers in Japan and demonstrated that corneal endothelial cell therapy was able…
In an October 19 news release from biopharmaceutical company Dicerna Pharmaceuticals, Inc. ("Dicerna"), the company shared results from the Phase 1 PHYOX4 clinical trial. During the trial, researchers sought to…
CRISPR (clustered regularly interspaced short palindromic repeats) was discovered over a decade ago by Emmanuelle Charpentier and Jennifer Doudna, two scientists who recently shared the Nobel Prize. Scientists using…
Let us begin this article recently published in MedCity News by starting with the ending: in order to help millions of rare disease patients waiting for rare disease treatments, a…
Early diagnosis is critical in many rare diseases, as earlier intervention leads to better treatment outcomes. Now, diagnosis is becoming easier and quicker through the use of artificial intelligence (AI).…
The COVID-19 pandemic has led to many changes, disruptions, and setbacks. For rare diseases, it did delay research for new treatments. However, there were a few positive things that came…
According to a recent article in Yahoo News, unless treatment is discovered or a cure is found for a disorder called IRF2BPL, these young children will lose the ability to…
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.
