Gonzolo and Jennifer Garcia were so excited when they learned that they were pregnant with their son, Asher. But during the 20-week ultrasound, they found out that Asher would be…
Branson Figueroa is four years old – and he’s tough. He has to be; after being born with craniosynostosis, Branson has had to undergo skull reconstruction surgeries twice already. According…
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…
According to a story from MSN, Joe McCauley was diagnosed with Leigh syndrome, a rare disease, last year. Now 30 years old, he was fortunate to survive a bout of…
According to the Institute of Cancer Research (ICR), a collaborative pilot study spearheaded by the Institute found that liquid biopsies could aid in the management, care, and treatment of…
According to a news release from the U.S. Food and Drug Administration (FDA) in early November 2022, Dupixent (dupilumab) is now approved for the treatment of adults living with…
Unfortunately, there are no cures - nor effective treatment options - for individuals with P-subtype small cell lung cancer (SCLC). This subtype is notoriously treatment-averse; it is also relatively…
This may sound like a strange title so let me explain. I live with a health condition called dystonia, which is a neurological movement disorder. It is the third most…
Laura Brown never planned on running more than one marathon. In 2012, she tackled a marathon as a way to cross an item off of her bucket list. But since…
When the COVID-19 pandemic began, many people wondered how it would impact them, especially if living with a rare disease or condition. As the pandemic has progressed, more research has…
Unfortunately, not much is understood still about chronic traumatic encephalopathy (CTE). However, we do know that it has been linked to repeated head trauma or head injury, such as that…
According to a story from globenewswire.com, the biopharmaceutical company Mereo BioPharma Group plc has recently announced that the company's investigational therapeutic alvelestat has earned Fast Track designation from the US…
According to a news release on Yahoo! Finance, clinical-stage biotechnology company Freeline Therapeutics Holdings plc ("Freeline") shared that it had initiated dosing in the second cohort of the Phase…
Fostamatinib is a spleen tyrosine kinase (SYK) inhibitor recommended to treat ITP patients who have been unable to receive a sufficient response from other medications. TAVLESSE® (fostamatinib), developed by Grifols…
In January, Harrison Moseby began having shoulder pain that was interfering with his day-to-day life. As a father and a firefighter, Harrison knew that he needed to be functioning as…
Dr. Paul Monga, director of Pittsburgh’s Liver Research Center, and his team are studying cancer and liver regeneration. Dr. Monga recently published a study that finds activation of a medication…
In his life, Paul Green accomplished a lot. He coached the North Queensland Cowboys, who won the National Rugby League title in 2015. Paul played for five different National Rugby…
This story was originally published by the Family Heart Foundation, a Patient Worthy partner organization. When Jessica’s father died of a heart attack at the young age of 54, she…
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…
Due to varied periods of decline among patients, previous research indicates that it is difficult to develop treatment options for patients who have relapsed DLBCL. This holds true especially when…
Patrick Freyne recently interviewed three people with myalgic encephalomyelitis (ME) for his column in the Irish Times. Patrick describes the circumstances behind the first known cases of the ME…
According to a late September 2022 news release from biopharmaceutical company AEON Biopharma, Inc., positive topline data is available from a clinical trial evaluating ABP-450 for patients with cervical…
On October 20, 2022, the Rare Disease Legislative Advocates (RDLA) hosted their monthly webinar. These webinar help provide updates to the rare disease community on legislation and other policy initiatives…
Three of Edith Lemay and Sebastien Pelletier’s four children were diagnosed with retinitis pigmentosa, an untreatable genetic condition. The parents were told their children would eventually be blind. In trying…
Master Chef Christine Ha of Dublin is coping with the fact that an accurate diagnosis years earlier would have prevented her blindness. Christine now spends a great deal of time…
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