Novartis AG, with corporate headquarters based in Basel, Switzerland, recently announced positive results from its Phase III trials REMIX-1 (NCT05030311) and REMIX-2 (NCT05032157) ). The news was delivered via Drugs.com.…
Previously approved for the treatment of narcolepsy, pitolisant (marketed under the brand name Wakix) may soon be leveraged for another rare condition: idiopathic hypersomnia (IH). Pitolisant is a histamine 3…
INT230-6, developed by clinical-stage biotechnology company Intensity Therapeutics (“Intensity”), is an investigational therapy comprising three moieties: cisplatin, SHAO-FA (a penetration enhancing molecule), and vinblastine sulfate. A moiety, in this sense,…
According to a story from PR Newswire, the pharma company Medivir AB, which is focused on developing cancer therapies in areas of critical high unmet need, recently announced the release…
Social media has become a vehicle for customer complaints and the companies are listening. Estimates are that almost two-thirds of dissatisfied customers have received a response to their online…
Also known as Richter’s Transformation, Richter syndrome is a rare and aggressive complication of chronic lymphocytic leukemia (CLL). When someone develops Richter syndrome, their CLL can transform into another rare…
Acknowledgment: This story is sponsored by Day One Biopharmaceuticals and is promoted through the Patient Worthy Collaborative Content program. We only publish content that embodies our mission of providing relevant,…
A team of surgeons at the Langone Institute performed its fifth animal-to-human transplant using organs from genetically modified pigs. The Revivicor company modified the organ in order to prevent rejection…
According to a study published on March 22, 2023 in the scientific journal Frontiers in Endocrinology, the rare genetic disorder X-linked hypophosphatemia (XLH) has a negative impact on the function of…
The International Congress of Parkinson's Disease and Movement Disorders took place this year from August 27 to 31. During the Congress, researchers and other stakeholders from across the globe…
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 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…
When a family member is unable to care for themselves, proper training is essential for all parties involved. A case in point was the crisis Patti LeFleur was unable…
65 million people in the U.S. who are on Medicare are prescribed medications according to a mid-year KFF healthcare survey. 60% of these individuals are prescribed a minimum of…
NOTE: The article was originally published as a blog post from the BHD Foundation a nonprofit patient advocacy organization. Birt-Hogg-Dubé syndrome (BHD) is a rare inherited condition associated with changes…
Teegan is Kelly and Matt Olson’s youngest daughter, so when she began missing developmental milestones, her parents were on alert. After a variety of tests over the span of…
Accutar Biotechnology, Cranbury, New Jersey recently announced via BioSpace that dosing has begun with the first patient enrolled in the Phase 1 trial of AC0676 a chimeric degrader molecule…
According to a story from Healio, a recent study doesn't bode well for people living with hidradenitis suppurativa (HS), a serious and debilitating skin disease. The data found that comorbidities…
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…
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…
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