370 words (source - 3% match) vs. 496 words (mine - 2% match) GEENIE can be used to develop in vitro and in vivo delivery mechanisms for drugs, gene editing…
The International Pemphigus and Pemphigoid Foundation is holding its 2023 Patient Education Conference from October 27-29, 2023. Since 2020, this conference has been held virtually online, and this format will…
The ocean is my favorite place in the entire world. When I see it—its immensity, its beauty—I feel immediately at home in the world. I believe that everyone should have…
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…
Jimmy Buffett survived a 1994 plane crash that was the inspiration for his song “Live Like It’s Your Last Day." According to an obituary on his website, Jimmy, 76,…
According to a story from Pharma Times, the biopharma company EsoCap has recently announced that it has finished enrollment for its phase 2 clinical trial. The trial includes patients from…
According to a story from The People's Pharmacy, a Senate report from 2018 highlights how opioid manufacturers regularly donate large sums of money to professional organizations and patient advocacy groups.…
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…
As reported by MSN, researchers have discovered that the blood vessels feeding aggressive brain tumors can be useful. The blood vessels have receptors that contain nanoparticles that can be…
The Phase 3 LITESPARK-005 trial sought to understand whether Welireg (belzutifan) was effective in treating advanced renal cell carcinoma (RCC) when compared to other treatments, such as everolimus. According to…
This year, September 17-23 is being recognized as Balance Awareness Week. Organized by the Vestibular Disorders Association, a Patient Worthy partner organization, the goal of this campaign is to help…
The month of September is recognized as National Sickle Cell Awareness Month in the US. This is a time to spread awareness about sickle cell disease among the general public…
In the United States, the FDA grants Orphan Drug designation to drugs or biologics that are intended to treat, diagnose, or prevent rare conditions. A rare condition is one…
Retinoblastoma is a rare cancer that begins in the retina, or the sensitive lining in the back of your eye. While it can affect people of all ages, it is…
245 words (source - 6% match) vs. 386 words (mine - 4% match) In the European Union, Orphan designation is granted to therapies intended to treat or use for a…
The 2023 Annual Meeting of the Myotonic Dystrophy Foundation took place from September 7-9 this year. During the meeting, stakeholders gathered in the nation's capital to discuss research, trends,…
As many people within the rare disease community know, a rare disease diagnosis can come with a significant financial burden. Additionally, the costs, treatments, equipment, and other needs are not…
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…
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