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A recent article from SciTechDaily describes the promising approach instituted at a private, non-profit biomedical research firm in San Francisco that incorporates data science, AI, stem cell biology, CRISPR, and…
Milenia Makani is an abstract artist living in London. She lives with Hypermobile Ehlers-Danlos Syndrome (hEDS), and has launched an international art fundraiser to support medical research through the Ehlers-Danlos…
A recent article by Dr. Rosalie Greenberg explores the possible relationship of infections caused by ticks and psychiatric conditions in children, including PANS or PANDA which are sudden onset. For…
Biossil, based in Toronto and co-founded by Dr. Alexander Mosa (Chair, CSO and co-founder) and Anthony Mouchantaf (CEO and co-founder), is making headway for rare diseases. Biossil’s core model is…
This, year the PCBers will meet in Indianapolis, Indiana. They will be celebrating 30 years as a community, as well as celebrating a much brighter future with the rare, autoimmune…
A recent article from Inside Precision Medicine states: For decades, systemic lupus erythematosus (SLE) has resisted tidy solutions, a shapeshifting autoimmune disease that crosses into different organ systems, flares without…
A New Shield for the Blood Supply: Grifols Earns FDA Approval for Malaria Test Grifols has reached a significant milestone in transfusion medicine with the FDA approval of its Procleix…
Yoga is often celebrated for its ability to help people find their "zen," but new research shows it might be doing some heavy lifting for your heart, too! A fresh…
As reported on Inside Precision Medicine, Researchers at Stanford have created a new urine test that helps doctors understand how well bladder cancer treatments are working. Usually, bladder cancer is…
On February 23, 2026, FDA issued a Draft Guidance document, called “Considerations for the use of the Plausible Mechanism Framework to Develop Individualized Therapies that Target Specific Genetic Conditions with Known Biological…
While surgery has long been the standard for removing solid tumors, a revolutionary technique called histotripsy is offering an alternative, as reported on MedPage Today. Histotripsy uses high-intensity, focused ultrasound…
The Dravet Syndrome Foundation of Spain hosted its 2026 Annual Family Meeting on March 20 at the Rafaela Ybarra School in Madrid, bringing together roughly 100 family members, researchers, and specialists.…
For families affected by phenylketonuria (PKU), newborn screening can mean the difference between a healthy future and lifelong complications. PKU is a rare inherited metabolic disorder that leads to toxic…
For people living with Lynch syndrome, the threat of cancer is a constant shadow. This inherited condition affects about one in 300 people, making them up to 80% more likely…
CureDuchenne is hosting several free events in 2026 to support families living with Duchenne and Becker muscular dystrophy. These events, called CureDuchenne CARES, will take place across the country. At…
Brussels — On February 24, 2026, the acclaimed Netflix documentary The Remarkable Life of Ibelin received the Media & Awareness Raising Award at the EURORDIS Black Pearl Awards. The honor…
As reported on InsidePrecisionMedicine, a new gene therapy aimed directly at the cells responsible for myelination is showing unprecedented promise for children with Canavan disease, a rare and fatal leukodystrophy.…
Madrid, December 3, 2025 - The research laboratory of the Dravet Syndrome Foundation was one of the initiatives awarded 3,000 Euros in the 11th edition of the Somos Pacientes ("We…
Author's Note: Patient Worthy began following Natera following their presentation at the American College of Medical Genetics conference many years ago. Natera has been known for its advances in prenatal…
Genetics: a cosmic role of the dice that plays a role in so many medical conditions, including bladder cancer. Mutations in FGFR3, PIK3CA, KDM6A, and TP53 are commonly associated with bladder cancer, but…
Lord Julian Fellowes, the Oscar-winning creator of Downton Abbey, has undergone a transformative treatment for essential tremor (ET) using Insightec’s magnetic resonance-guided focused ultrasound (MRgFUS) technology. After living with the…
Each year, Patient Worthy covers the American College of Medical Genetics conference, the leading source of both genetic education for health care professionals and a leading source of new information…
Pancreatic cancer took Alex Trebek, Michael Landon, Ruth Bader Ginsburg, Luciano Pavarotti, Steve Jobs, Patrick Swayze, and my dear friend Amy. Glioblastoma (GBM) took Senators John McCain and Ted Kennedy,…
Editor's Note: Patient Worthy is proud to share this list of resources for those living with stomach cancer. No Stomach for Cancer- This organization has a global reach and specializes…
Genetic testing is now more available than ever before. For those who suspect a genetic disorder, the best route is often through a genetic counselor. They can advise you not…
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