A groundbreaking clinical milestone has been reached in gastroenterology with the publication of a first-in-human study evaluating a novel spray gel for the treatment of ulcers following endoscopic submucosal dissection…
In a remarkable stride toward personalized cancer treatment, scientists have developed a groundbreaking test designed to predict whether individual patients will respond to chemotherapy or develop resistance. According to Bioengineering.com,…
LA Jolla Institute (LJI) scientists have recently discovered that T-cells, known primarily as disease fighters, may also play a role in the development of Parkinson’s disease. The researchers, working in…
Researchers at the University of California, Davis, have developed an investigational brain-computer interface (BCI) that holds promise for restoring the voices of people who have lost the ability to speak…
A recent article from PMLiVE underscores the critical role of collaboration in advancing innovation and improving access to treatments for rare diseases. With over 7,000 rare diseases affecting millions of…
A recent article from Pharmaceutical Manufacturer highlights a significant achievement in the rare disease sector, as Rare Disease Biotech announced reaching a major milestone in the development of its new…
A recent article published in the Orphanet Journal of Rare Diseases highlights the launch and goals of RARE-Impact, a new European project designed to enhance the diagnosis, care, and overall…
Researchers have discovered a potential “window” whereby babies born with inherited diseases may be treated with gene therapy sent directly into their circulatory systems. The window consists of circulating stem…
A recent study has revealed that vitamin B3 could offer hope for patients suffering from a rare genetic disorder that causes accelerated aging. As reported by ScienceBlog, researchers found that…
The FDA recently informed Muscular Dystrophy News that it has granted orphan drug designation to the experimental drug ISX9-CPC, a product of the IPS Heart company located in Houston, Texas.…
Applied Therapeutics has encountered a significant setback in its quest to bring a new treatment to patients with a rare metabolic disorder. According to Biospace.com, the company announced that its…
An article titled “Accelerated Approval as the New Norm in Gene Therapy for Rare Diseases” from DIA Global Forum digital magazine’s May 2025 issue explores the evolving landscape of regulatory…
This week, the FDA approved the first blood-based diagnostic test for Alzheimer's disease (AD). The test, known as "Lumipulse G pTau 217/β-Amyloid 1-42 Plasma Ratio," is designed to detect amyloid…
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.…
Immune dysfunction and inflammation have been found to play significant roles in Alzheimer's disease. An analysis of brain tissue in an animal model reveals a potential for immune system dysfunction at…
The use of the gene therapy elivaldogene autotemcel (Skysona), has been approved due to its success in the treatment of patients diagnosed with the neurological disease cerebral adrenoleukodystrophy (CALD). The…
According to a recent report in Healio News, the GLP-1 agonists are medications developed primarily to control blood sugar levels for type two diabetes. About the FLOW Clinical Trial The…
Patient Worthy's award-winning podcast "Wait, How Do You Spell That? A Rare Disease Podcast" is back with a new episode. On this episode of the podcast, we discuss the role…
The Riken Center team, headed by Dr. Takaomi Saido and using mice with Alzheimer’s, discovered that dopamine may lessen the brain’s physical symptoms and improve memory. The study, featured in…
According to a story from Healio, a recent study determined that adults living with Graves' disease who were being treated with antithyroid medications will have an elevated risk of developing…
A protein called Aplp1 is now on the radar for its ability to spread material in the brain that may be responsible for Parkinson’s disease. Another protein, Lag3, interacts with…
According to a story from Healio, analysis of the FDA Adverse Events Reporting System database indicates that tirzepatide has a superior safety profile when compared to GLP-1 inhibitors that have…
This article was written by and shared with permission from Shannon Wieloch, MS. Shannon is a certified genetic counselor and patient advocate for the Aicardi-Goutiere Syndrome Advocacy Association and founder of Stork Genetics.…
Researchers discovered a version of the Cas protein that fits into adeno-associated viruses (AAVs). This virus can be engineered to deliver targeted therapy to cells and is a common approach…
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