Ally Winter was diagnosed with short bowel syndrome when she was four years old. Now, she is 17 years old and working to raise awareness for rare and chronic illnesses.…
KRYPOLIS (carfilzomib) recently received conditional approval in China for the treatment of relapsed/refractory (R/R) multiple myeloma. Licensed by BeiGene, this drug is indicated for adults with R/R multiple myeloma who…
Medical professionals are still working to uncover the cause of Alzheimer's disease. While they believe that a combination of genetic and environmental factors paired with certain lifestyle choices leads to…
Accessing treatments can be difficult, especially if one is impacted by a rare disease. In fact, only 5% of rare diseases have an FDA-approved treatment. Because of this, many patients…
According to MedPage Today, the FDA sent a letter to the manufacturer of the Johnson & Johnson COVID-19 vaccine on Monday containing a warning about its link to Guillain-Barre syndrome…
The CDC is currently looking into three cases of melioidosis throughout the United States, according to an article published in MSN. This rare, bacterial infection is mainly found in the…
We know that it can be difficult to receive the correct diagnosis for a rare disease. Sometimes, you have to be your own advocate. This means learning everything you can,…
Amyotrophic lateral sclerosis (ALS) is a rare, neurological disorder that is characterized by the progressive death of nerve cells and muscle weakness. As time passes, many affected individuals experience difficulty…
Rare diseases face many obstacles when it comes to research and drug development. Small patient populations, a lack of awareness, and limited funding are just a few examples. When it…
We know that COVID-19 leaves some with long-lasting symptoms. In fact, we are beginning to discover the connection between the respiratory virus and chronic fatigue syndrome (CFS). Now, CFS patients…
Newborn screening is important to early diagnosis and intervention. With some conditions, every second counts. Catching a rare disease as soon as a child is born could lead to much…
The FDA grants designations, such as the Rare Pediatric Disease and Orphan Drug designations, to aid in the drug development process. Recently, LEXEO Therapeutics has received these two designations for…
It's summertime, and that means tick season is upon us. We've all had to deal with the annoying insects, whether it's pulling them off our pets, spraying bug spray before…
We're learning more and more about COVID-19 every day. One thing that we're discovering as the pandemic continues is the complications that may arise after recovery. Chronic fatigue syndrome, brain…
People are rushing to get in shape for summer; it's common to see the gyms a little more full than normal. While exercise is good for you, it's important not…
Medical professionals have been searching for a viable treatment for Parkinson's disease for a long time. While there have been advancements and new therapies created, many patients are still looking…
Federal officials from the CDC and FDA have recently come out to address the possible connection between mRNA vaccinations for COVID-19 and cardiac issues. This recent statement acknowledged that there…
A two-month-old baby recently traveled to Dubai to receive treatment at Al Zahra Hospital Dubai (AZHD), according to an article in the Khaleej Times. The child, who is from Oman,…
Mark Dalziel has big plans for this weekend; he's going to bike the length of Scotland within two days. He has a very good reason for doing so, which is…
The National Medical Products Administration (NMPA) of China has recently approved surufatinib for drug registration for the treatment of advanced pancreatic neuroendocrine tumors (pNETs). HUTCHMED Limited recently announced the news,…
We're still trying to understand COVID-19, and it may take us years and years to fully comprehend the respiratory virus. A recent case study published in the Canadian Medical Association…
You may know Jessie J from some of her hit singles like "Price Tag" or "Bang Bang," but did you know that she is also a rare disease patient? She…
Rare disease patients often face more obstacles than others when it comes to healthcare. While we know this, it can be hard to judge the public's stance on the issue.…
The Quincy community is helping to organize a bone marrow registry drive for a local teen who was recently diagnosed with hemophagocytic lymphohistiocytosis (HLH), according to an article in The…
We still don't fully understand a number of rare diseases. A lack of awareness, lack of interest, and lack of funding all play a role, and medical professionals simply can't…
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.
