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The University of Michigan has opened a $10 million research center dedicated to histotripsy, a technique that uses focused sound waves to destroy tumors. The center will make specialized equipment…
Fabry disease is a rare inherited condition caused by changes in the GLA gene. These changes can lead to low or absent activity of an enzyme called alpha-galactosidase A. As…
IndoUSrare and GORD Foundation are honored to announce the 2026 Abbey Meyers Khushi Bridging RARE Award Honorees: Rare Disease is a global problem, a human problem, and the individuals below…
Two papers published in Nature Communications report new findings about roginolisib, an experimental cancer drug being developed by iOnctura. One study describes how the drug interacts with a cancer-related enzyme…
As reported on Inside Precision Medicine, a novel optogenetics-based treatment may offer a new path toward restoring some visual function in people with advanced retinitis pigmentosa (RP), according to findings…
Aspire: “ We aspire to improve the lives of children and adults affected by PANS/PANDAS and other immune-related encephalopathies by closing the gap between symptom onset and access to treatment…
Two-year University of Arizona study will use human brain-cell models to investigate sex-specific biology and help guide future research and clinical trials. Critical Path Institute (C-Path) and the University of…
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…
As reported on SciTechDaily, a large research review suggests age-related losses in muscle mass, strength, and physical function may have a stronger association with dementia risk than obesity alone. Maintaining…
Diabetic retinopathy affects approximately 30% of adults with diabetes and stands as one of the leading causes of vision loss among working-age individuals worldwide. The condition damages the delicate blood…
People who inherit a rare change in the EGFR gene may be much more likely to develop lung cancer. Their risk is about 25 times higher than that of people…
Patients in the United States wait an average of 34 to 50 days (roughly 1 to 1.5 months) for an initial neurology appointment, with wait times stretching up to 4…
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…
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