A court in Zabbar, Malta found the parents of seven-year-old Victoria guilty of her death by negligence. The court also determined that the system had failed the child by…
A positive opinion is needed from the Committee for Medicinal Products for Human Use (CHMP), part of the European Medicines Agency (EMA), to begin marketing a drug. Once a positive…
Ticks can carry a number of disease-causing pathogens. If an infected tick bites a human, that human can then contract several illnesses. In many cases, people who contract these…
In Japan researchers at the Juntendo University have discovered that when ECHS1 enzyme variations of mitochondrial enoyl-CoA hydratase short chain 1 (ECHS 1) do not function properly, they cause…
WebMD recently carried a story about a young woman who, after nine years of wheelchair confinement caused by a rare metabolic disease with no name, was able to walk…
The FDA has approved the drug Ayvakit developed by the biotechnology company Blueprint Medicines for the treatment of indolent systemic mastocytosis. According to an article in BiopharmaDive, Ayvakit had been…
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…
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…
As clinicians and scientists continue to delve into RNA sequencing benefits, they are learning how RNA sequencing provides insights for people with rare genetic conditions. The learning experience will provide…
On May 18, 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…
Fierce Biotech recently carried an article about the death of Terry Horgan. Terry was a 27-year-old Duchenne muscular dystrophy (DMD) patient and the brother of the CEO of the…
The 91st European Atherosclerosis Society (EAS) Congress took place from May 21-24, 2023. During the Congress, many stakeholders discussed the latest developments in basic, translational, and clinical research into vascular…
The energy in the crowd at American Family Field was electric. Everybody sat on the edge of their seat, waiting for the game to begin. If you’ve never been to…
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…
John knew from an early age that he not only loved music but had confidence in his musical ability. However, there was another very troubling issue in his life. John…
In a late May 2023 news release from clinical-stage biotechnology company Acepodia, the company shared that the first patient has been dosed in a Phase 1 study. Within this…
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…
Managing dystrophic epidermolysis bullosa (DEB) can be difficult. DEB is a rare genetic condition which causes fragile, easily blistered skin. There are multiple DEB subtypes with their own inheritance…
An announcement from the drug company Genentech highlights the results of a recent study which demonstrated that the drug faricimab-svoa (marketed at Vabysmo) was able to dry retinal fluid more…
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…
Ventilatory assistance, NG tubes, tracheostomy: all of these may be used to care for infants born with bronchopulmonary dysplasia (BPD), a chronic lung disease that most commonly affects preterm…
Unfortunately, there are a number of negative or false societal narratives around the concept of “rare disease” or “disability,” particularly when it comes to what these individuals can achieve. Having…
In December 2022, Liam Hendriks—a pitcher for the Chicago White Sox—was diagnosed with non-Hodgkin’s lymphoma (NHL); he shared this news with the world in January 2023, which Patient Worthy reported…
As reported in Yahoo! Finance, the United States’ Food and Drug Administration (FDA) recently granted Orphan Drug designation to MP1032. This therapy, developed by clinical-stage biotech company MetrioPharm, is being…
At first, the round black dots that Carmen saw spattered across the bathroom sink were frustrating, but not concerning. Since her grandchildren were staying with her, Carmen assumed that something…
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