Collaboration has always made the world go round - and it's no different in rare disease research. Sometimes, the community affected by a certain rare disease is small, which…
When it comes to staying active, 8-year-old R.J. Walters has it covered. His favorite sport right now is basketball. But that hasn’t stopped R.J. from getting his yellow belt in…
Dear fellow rare community, It is with great joy that I share my journey of self-discovery and self-love with you. It took me a while to fully embrace my uniqueness…
Altogether, there are an estimated 300,000 people in Ireland who are living with a rare disease. However, given the increasing amount of genetically-oriented rare diseases, it's possible that even…
On April 20, 2023, the Rare Disease Legislative Advocates (RDLA) hosted its monthly webinar. These webinars help provide updates to the rare disease community on legislation and other policy initiatives…
In a news release on the company’s website, biopharmaceutical company Applied Therapeutics, Inc. shared that positive data was available from the Phase 3 ACTION-Galactosemia Kids trial. Within the trial, researchers…
Rylee Riekena feels most at home when she’s near water. There’s something about the ocean that just makes her feel safe and at peace. So when she took up paddleboarding,…
Rare Community Profiles Rare Community Profiles is a new Patient Worthy article series of long-form interviews featuring various stakeholders in the rare disease community, such as patients, their…
Each year, students at Carteret Community College in Morehead City, North Carolina participate in the “Great Strides” Walk for Cystic Fibrosis. “Great Strides” is the Cystic Fibrosis Foundation’s largest fundraising…
Over the years, there has been minimal improvement in the life expectancy differences between white and black people living in the United States. Yet, according to an article featured…
According to a recent study from McGill University in Canada, the new method for classification of brain illnesses has been identified. This study was led by Yashar Zeighami and compared…
On February 1, 2023, FDA issued a Draft Guidance Document, called “Considerations for the Design and Conduct of Externally Controlled Trials for Drug and Biological Products” (Docket ID: FDA-2022-D-2983). The Draft Guidance considers…
On April 12, 2023, Research!America, a Patient Worthy partner organization, hosted a webinar program titled Better Data for More Equitable Public Health Research. This talk featured Dr. Mary Pittman, President…
When Alexander Barron (9) was first born, doctors told his parents that he would likely never walk. In fact, said doctors, Alexander would probably not be very independent at all…
For as long as he can remember, Geoff Rhyne has loved food - especially when he gets to cook it. After all, it was this passion and creativity that led…
Porphyria refers to a group of rare disorders that cause issues with the production of heme, which is part of what makes up hemoglobin (a protein in your red blood…
The first few years of Saylor Baysden’s life involved a good deal of medical confusion. Her family pursued testing, leading to two diagnoses in 2020-21: autism and epilepsy. But her…
When the man in his 60s first hurt his leg, he wasn't too worried about it. He had cut his leg open on the door to his caravan and, like…
Children all across the globe enrolled in a study to evaluate Upstaza, a gene therapy medication, for the treatment of aromatic L-amino acid decarboxylase (AADC) deficiency. As part of their…
Medical research is crucial in better understanding diseases and discovering novel treatment options. Unfortunately, there is often one large barrier to research: funding. This is especially true within the rare…
The National Institutes of Health (NIH) held its in-person Rare Disease Day at NIH convergence on February 28, 2023. This was the first time in a while that the event…
Reanna and Malaky always knew they wanted to start a family together. They tried for a while without success—so they were thrilled when they learned that Reanna was pregnant with…
According to a press release from the US Food and Drug Administration (FDA), on March 24, 2023, the agency issued a new draft guidance titled Clinical Trial Considerations to Support Accelerated…
The Higbee family never planned to become rare disease advocates. But when their daughter Harlow began experiencing health issues, they knew that they would do whatever they could to help.…
When Sue Hitchmough first began feeling ill, she wasn’t sure what to think. Doctors told her that she had an ear infection, but antibiotics didn’t seem to fix anything. She…
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