As reported in Biospace, Taysha Gene Therapies wants to eradicate devastating genetic epilepsies and central nervous system disorders rooted in a single genetic mutation. That’s going to take some innovative…
Biotechnology company Stealth Biotherapeutics ("Stealth") has made the company's mission to develop and commercialize treatments for patients with diseases characterized by mitochondrial dysfunction. One leading treatment in the company's…
According to a story from Sano Genetics, Sano Genetics, BenevolentAI, C4X Discovery, and Patients Know Best expect only the most from their novel collaboration. Together, they are pooling top expertise…
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…
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…
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,…
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…
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…
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…
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…
According to a story from wavy.com, Cherie Reenee Brown was a prominent political organizer and activist in the city of Portsmouth, VA. But on New Years Day 2021 she passed…
A survey created by Eurodis, a European rare disease advocacy alliance, has shown that those living with a rare disease have a worse healthcare experience than people with a chronic…
Every year for the past six years the National Organization for Rare Disorders (NORD) has published a State Report Card on the progress of rare disease policies in the states.…
As reported in a press release in PR Newswire; in Stockholm this week, Dan Kastner received the Crafoord Prize, one of the top achievements in science worldwide, after his work…
Tom Kean, the Senate Republican Majority Leader in New Jersey, is leading the charge to fully realize a recently partially approved bill, which would create a New Jersey Rare Disease…
Dr. Oliver Dorigo of the Stanford University Medical Center was the medical source for an article featured in Oncology. Dr. Dorigo explained that if a woman with ovarian cancer has…
Genethon and WhiteLab Genomics have entered into a partnership agreement, which will allow them to further develop their work in artificial intelligence and gene therapy. Within the terms of the…
Michael Freeland spent most of his life trying his best not to think about his incredibly rare disorder. Not only was it incredibly painful, but the symptoms were debilitating, and…
Patient groups can optimize patient registries, cross-sectional studies, and/or longitudinal natural history studies to maximize collaborations with sponsors of orphan drugs and gene therapies. Written by Harsha Rajasimha, MS, PhD…
The United Kingdom has taken a big step in the right direction for rare disease patients, as they have recently created the UK Rare Diseases Framework. This framework is intended…
Hundreds of mutations have been connected to autism spectrum disorder (ASD), and a new study finds further evidence that suggests a possible cause of this spectrum of conditions Neuroscience News…
The COVID pandemic affecting eighty-million children in the nation has been a burden on many families. A number that deserves attention is the seven million students who have learning disabilities.…
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…
On her 23rd birthday, Sara Lefebvre almost died. After having some drinks with friends, her friend drove Sara home. But Sara didn't make it inside; she collapsed in sub-zero temperatures,…
Last year, 92.7 WOBM reporter Vin Ebenau sat down with Allie and Jonna Crocker, two sisters who live around the Jersey Shore, to discuss their experience with rare diseases. The…
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