.
As reported on MedPage Today, an experimental immunotherapy called gotistobart helped patients with advanced squamous non-small cell lung cancer (NSCLC) live longer than standard chemotherapy in the phase III PRESERVE-003…
“For people living with a progressive rare disease, time is measured differently. Research and clinical trials can take years while patients continue to lose strength and function,” said Kathryn Bryant…
One-time treatment provides a new option for pediatric patients with a rare, progressive genetic disorder As announced in a recent press release, the U.S. Food and Drug Administration has approved…
The condition Dupuytren disease, also called Dupuytren contracture, is a chronic disorder of connective tissue in the palm. Tissue gradually becomes thickened and tight, forming cords that can pull one…
What's the Good News? Researchers have successfully completed initial testing of an experimental Parkinson's medication called TT-P34. The early results are very encouraging and suggest the drug is safe, works…
The history of cystinosis is one of the most successful examples of rare disease research leading to meaningful treatments. In a recent interview with the European Medical Journal, William Gahl—a…
A single genetic change can have dramatically different effects from one person to the next. That is the reality for people living with PTEN hamartoma tumour syndrome (PHTS), a rare…
As reported on Inside Precision Medicine, a new, non-invasive test may help doctors detect oral cancer quickly and reduce the need for painful scalpel biopsies. In a large study involving…
A Sustainable Biologic Solution Gains Major Insurance Coverage and Proves Superior to Synthetic Alternatives in Real-World Clinical Data In the world of advanced wound care and reconstructive surgery, an unlikely…
RASONQUE Represents a Watershed Moment for a Disease That Has Resisted Treatment Since RAS Was Discovered as a Cancer-Causing Gene Pancreatic cancer is among the cruelest malignancies. It develops silently,…
A Rare Disease Gets Its First FDA-Approved Medicine—And It Works Differently Than Anything Available Before Imagine your immune system getting the signals completely wrong. Instead of protecting you from infection,…
Today the Tin Soldier Disease has a treatment. A clinical trial showed a 90% reduction in new bone development in adults with FOP. After Decades of Hope Deferred, Pasatru Offers…
Researchers Discover That One of Medicine's Most Common Lab Tests May Warn of Rare But Serious Side Effects Weeks Before They Occur As reported on Inside Precision Medicine, CAR-T cell…
Scientists Discover Multiple Pathways Through Which an Ancient Fruit Protects the Cardiovascular System For centuries, pomegranates have held a special place in Middle Eastern and Mediterranean cultures—prized not just for…
The Basic Problem: Why One Bad Gene Can Cause Big Trouble Imagine your body as a giant factory. Like any factory, it needs quality control—machines that make sure good products…
Historic Milestone Offers Relief from Relentless Burden of Glycogen Storage Disease Type Ia According to a recent press release from Ultragenyx, after decades of medical innovation and tireless advocacy from…
What Happened? Hansa Biopharma, a Swedish company, announced that the first patient in Australia has successfully received a kidney transplant after being treated with a new medication called Idefirix® (imlifidase).…
As shared on GlobeNewswire, a biotechnology company called ABLi Therapeutics has released encouraging findings from a research study on a potential new Parkinson's treatment called risvodetinib. Here's what you need…
According to a press release from Texas A &M, researchers are using artificial intelligence to narrow drug choices and make shared data easier to use. Tuberculosis drug discovery speeds up with new…
Coats disease, is a rare retina condition diagnosed primarily in children. Symptoms may include, eye drift, blurry vision, the appearance of a white or silver looking retina, and retina detachment.…
As reported on Inside Precision Medicine, scientists have developed a way to spot a specific lung cancer mutation in just minutes instead of weeks, using a combination of advanced laser…
Medical device innovator Xeltis has officially kicked off the European commercial rollout of its novel vascular access implant, aXess™. The milestone was marked by the first routine commercial procedure, performed…
The pharmaceutical company Grifols—has started dosing patients in the final stage (Phase 3) of two clinical trials. The company is trying to get approval to use two of its antibody-based…
As reported on Inside Precision Medicine, for years, a chronic spinal cord injury (SCI) has felt like a permanent biological roadblock. When the spinal cord is damaged, the immediate aftermath—known…
New research from the 2026 American Society of Clinical Oncology (ASCO) annual meeting, and presented on Cure Today, highlights an encouraging new treatment on the horizon for advanced non-small cell…
© Copyright 2024 Patient Worthy
Sign Up With a Patient Worthy Account and Share Your Rare Story
- OR -
Make a difference, share your experiences and get paid. Opt-in and join Patient Worthy's panel for paid opportunities such as surveys, market research, patient advisory panels and more.
- OR -
Make a difference, share your experiences and get paid. Opt-in and join Patient Worthy's panel for paid opportunities such as surveys, market research, patient advisory panels and more.
