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On February 23, 2026, FDA issued a Draft Guidance document, called “Considerations for the use of the Plausible Mechanism Framework to Develop Individualized Therapies that Target Specific Genetic Conditions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…
Acknowledgement: Patient Worthy is honored to share this story by Erika Stariha, Co-Founder and President of SATB2 Europe. This is Urban, my teenage firstborn. He is joyful. Curious. Full of…
According to an article in Inside Precision Medicine Researchers have developed a new, easy-to-use test called SHINE-TB that can quickly diagnose tuberculosis (TB) using saliva samples. This test is based…
Researchers in Japan have developed a personalized mRNA vaccine that shows promise for treating recurrent gastric cancer, specifically peritoneal metastasis, which is often difficult to manage with existing therapies. This…
Kevan Chandler, was born with a rare, and progressive neuromuscular disease: spinal muscular atrophy (SMA). At age 33, he needs assistance with almost all activities of daily living, from eating…
Each year, nearly half a million Americans are affected by Lyme disease, a tick-borne illness that, if untreated, can lead to serious complications like arthritis, heart issues, and neurological disorders.…
Acknowledgement: Patient Worthy is honored to share this story from our friends at GACI Global. To view the original article, please click here. Sharon’s passion for children shines in her…
Danielle’s baby boy had a long and terrifying seizure on the 4th of July six years ago. The family was at her mother’s home. Both Danielle and her mother are…
Do you know about Lafora Disease? This rare genetic disease begins during adolescence. Your child starts their life healthy and whole, but then they decline, cognitively and physically, until they…
Myrovlytis Trust, a UK-based charity and Patient Worthy partner, has developed a new resource for those facing osteosarcoma. This devastating cancer often occurs in childhood, teen, and young adult years.…
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