Recently, clinical stage biopharmaceutical company Immunic, Inc. ("Immunic") has been working to develop potential treatment options for patients with primary sclerosing cholangitis (PSC), a rare condition for which no…
Research by scientists at Cornell University and Weill Cornell centered around the endothelial cells that form a barrier between tissues and vessels. They control the flow of fluid and substances…
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 the pandemic rages on, doctors across the country are beginning to notice a trend. A number of children who have contracted COVID-19 have also been impacted by a rare…
According to a story from ktvl.com, seven year old Emma Suetta, of Etna, California lives with a rare disease called cystinosis, and she is doing her part to help find…
If you’re a caregiver for a loved one with a rare disease, it’s not presumptive to assume you work tirelessly managing your loved one's busy appointment schedule, daily stretch routine,…
New research suggests that ultrasounds could be a new diagnostic tool for patients with a rare lysosomal storage disorder. According to Fabry Disease News, kidney dysfunction is characteristic in Fabry…
The NCCN, or National Comprehensive Cancer Network, has recently published treatment guidelines for children living with a rare kidney cancer, Wilms tumor. This is the organization's first guidelines written for…
According to a recent press release, scientists studying the Uniformed Services University of the Health Sciences' (USU) The American Genome Center (TAGC) identified five genes which could play a role…
Raghav is a two-year-old boy born with an ultra-rare disease caused by a mutated GPX4 gene. He and eight other children are the only ones in the world who have…
A study recently published in The FASEB Journal and led by Dr. Chiharu Nishiyama from Tokyo University of Science has demonstrated that a compound found in blueberries could serve as a…
For nearly one year now, COVID-19 has dominated our headlines. In 2020, news emerged of SARS-CoV-2, a virus causing a global pandemic. Now, in February 2021, there are 110 million…
Tonix Pharmaceuticals and Inserm, which is the French National Institute of Health and Medical Research, have entered into a licensing agreement in which Tonix will hold the license to oxytocin-based…
According to a story from The Whitewater Company, parents Paul & Heather Lynn were at a loss for what to do when their daughter Clarity was born with the rare…
Arteriovenous malformation (AVM) is a rare disease that causes blood vessels to clump together within the skull. It can lead to aneurisms, strokes, or early death. However, surgery is possible…
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…
As reported in Biospace, a collaboration between biopharmaceutical company AstraZeneca and BenevolentAI has for the first time used AI to identify a molecule that plays a key role chronic kidney…
Just over a week ago, drug discovery and development company Rescindo Therapeutics Inc. ("Rescindo") shared that its drug candidate RSC-57 received both Orphan Drug and Rare Pediatric Disease designations. The…
The last day in the month of February is recognized each year around the world as Rare Disease Day. The day serves primarily as a moment of spreading awareness about…
In the past, researchers have questioned the exact cause of spina bifida, a neural tube defect in which the spine does not fully close around the developing spinal cord nerves.…
Jason Kraft, the owner of Electric Bike Technologies, has brightened the lives of two sisters with two electronic tricycles that are typically made for senior citizens. Maggie and Charlie Monnin,…
According to Targeted Oncology, the first patient was dosed in a Phase 1 clinical trial evaluating NBTXR3, an investigational tumor-agnostic radio enhancer, for patients with esophageal cancer. The trial will…
Ocular Therapeutix has announced that they will attend and present at the Angiogenesis, Exudations, and Degeneration 2021 Meeting, which is being held virtually due to the pandemic. They intend to…
While gene therapy is a promising field, there are some associated risks: unwanted immune reactions, infections, or whether the therapy could lead to the development of other conditions, like cancer.…
Policymakers Must Offer Solutions That Provide Patients with Lower Out-of-Pocket Costs For Critical Medicine In addition to coping with chronic illness, many patients are faced with the challenge of…
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