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…
Dr. Alan Grant (portrayed by Sam Neill) began Jurassic Park with a distaste for computers and children, intent only on developing his paleontological prowess. By the end of the…
In a news release from mid-April 2023, clinical-stage biotechnology company Abivax SA shared that 2-year results were available from a Phase 2b open-label maintenance study evaluating obefazimod for individuals…
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…
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…
HealthReady has partnered with the Beckley Foundation for a virtual coffee break webinar program titled "Use Your Child's Health Data for Better Outcomes in Neuroblastoma." Neuroblastoma is a dangerous cancer…
According to a story from MedCity News, the drug company Eli Lilly was anticipating the approval and launch of its drug mirikizumab as a treatment for ulcerative colitis. However, the…
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,…
It’s no secret that, in many cases, funding for rare disease research and drug development is slim; the financial burden often falls to families and patients. 40 years ago, the…
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…
The passage of the Orphan Drug Act was an important step in creating an environment that supports rare disease research and drug development. This Act created Orphan Drug designation,…
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…
Walt McGrory always knew that he wanted to play basketball. So when he became a student at the University of Wisconsin-Madison (UW-Madison), he joined the Badgers as a walk-on. In…
According to a story from wmur.com, a 12-year-old boy named Grady Smith from Salem, NH lives with adrenoleukodystrophy, a rare disease. Grady is a big fan of basketball (his favorite…
Our blood has different clotting factors, or protein, that helps clot and stop bleeding after injuries. But when you don't have enough of these proteins, the blood can't clot…
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…
In February 2023, I spoke with Dr. Allen Davidoff of XORTX Therapeutics Inc. (“XORTX”) about the company’s commitment to developing therapies for people living with progressive kidney disease. We…
According to a story from BioPharma Dive, the drug company AbbVie has recently announced its decision to withdraw two different approvals for its cancer therapy Imbruvica. The approvals were withdrawn…
Champlain Valley Union (CVU) High School students are running on a high this season after the school's basketball team clinched its first ever state championship win. The CVU RedHawks…
In an article for WebMD, Marilyn Gentile discusses her experience with age-related macular degeneration. She had cataract surgery at 70 years old and just days later she had a central…
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…
People with rare undiagnosed conditions present challenges to doctors when they are attempting to understand the root causes of their patient’s health problems. A recent article in Medical Xpress reports…
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…
STAT NEWS recently published an op-ed with a provocative question: How do we tell the difference between legitimate mental health tech and snake oil? Looking back to the early…
Written by Salem Taylor My name is Salem, I’m 43 years old, live in North Carolina and have hyperkalemic periodic paralysis. Growing up as a tall, awkward home-schooled kid was…
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