On February 1, 2023, FDA issued a Draft Guidance Document, called “Considerations for the Design and Conduct of Externally Controlled Trials for Drug and Biological Products” (Docket ID: FDA-2022-D-2983). The Draft Guidance considers…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According to a press release from the US Food and Drug Administration (FDA), on March 24, 2023, the agency issued a new draft guidance titled Clinical Trial Considerations to Support Accelerated…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…
At Patient Worthy, we seek to partner with patient advocacy and other related organizations in order to collaborate and promote one another's activities. Recently, we have begun a partnership with…
When Harper Webb was just six weeks old, her mother Johnna grew concerned about the girl’s health—call it mother’s intuition. Harper struggled with sleeping and feeding. Johnna began taking Harper…
On March 23, 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…
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…
In 2022, only around 63 cases of ECHS1, an ultra-rare metabolic disorder, were reported around the globe. A study published in the Annals of Clinical and Translational Neurology in 2015…
The complexity of rare diseases makes communication between adults involved with the care of these children a vital tool. Communication and mutual trust are of the utmost importance. About the…
JScreen is a non-profit organization headquartered at the department of Human Genetics at Emory University in Atlanta. JScreen offers genetic testing and educational services, such as genetic counseling. JScreen's mission…
A mother knows best. In Kaitlyn Fryar's case, her intuition about her son Hudson led her to pursue help from numerous doctors. According to CBS Pittsburgh, Hudson seemed to…
On March 9-10, 2023, Dynamic Global Events hosted the virtual Global Innovation in Patient Advocacy conference. This event was geared towards various stakeholders in the rare disease space and was…
In many rare conditions, early diagnosis and treatment typically correspond with better outcomes. Prior to March 2023, LIVMARLI (maralixibat) was approved for the treatment of cholestatic pruritus related to Alagille…
The Mississippi State University (MSU) Bulldogs joined forces with Children’s of Mississippi, the state’s only children’s hospital with statewide pediatric clinics, to honor pediatrics patients throughout Mississippi, sharing and uplifting…
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