“We’re going to Disney World!” That's what all the winners say, right? When the big game is over and the team has won the championship, the winners say, for a…
In a recent statement, The U.S. Food and Drug Administration (FDA) has introduced the Technology-Enabled Meaningful Patient Outcomes (TEMPO) Pilot, a groundbreaking initiative aimed at expanding access to digital health…
As reported by MedicalXpress, Researchers have uncovered a surprising immune function in specialized intestinal cells that may reshape our understanding of gut health and autoimmune disorders. The study, published in…
A team of doctors at NYU Langone transplanted a pig kidney into a brain-dead patient and reversed its rejection twice during the 61-day study. Currently, estimates of the number of…
Breaking a system into small independent and interchangeable modules that can perform certain functions is called modularity. For many years the KRAS oncogene proved to be a classic example of…
Chiesi Group has entered an exclusive licensing agreement with Aliada Therapeutics, a subsidiary of AbbVie, to develop therapies that can cross the blood-brain barrier (BBB) for lysosomal storage disorders (LSDs).…
A new artificial intelligence model, popEVE, could accelerate diagnoses for rare, single-variant genetic diseases by scoring how likely each genetic change in a patient’s genome is to cause disease. Developed…
The findings were recently published in Nature Immunology and reported by Precision Medicine suggesting that a factor called Interferon Regulatory Factor 4 (IRF4) takes responsibility as a driver of transplant…
A gene-editing protein, small enough to fit into adeno-associated viruses (AAVs) that infect humans has been identified. As reported by Precision Medicine, these compact AAVs can be modified so that…
As reported on PharmaBiz, the National Organization for Rare Disorders (NORD) has announced a new request for proposals (RFP) aimed at nonprofit patient advocacy groups interested in launching rare disease…
Why Rare Diseases Pose a Diagnostic Challenge As discussed in the Orphanet Journal of Rare Disease, a "rare disease" is defined as a condition affecting fewer than 1 in 2,000…
Please go HERE to read Part 1 of this article The company, GEMMABIO, will pursue a number of rare, large indications. A new spinout, RareTx, will be dedicated to Orphan…
The U.S. Food and Drug Administration has approved UCB's Kygevvi, marking a historic milestone for patients with thymidine kinase 2 deficiency (TK2d), an ultra-rare inherited mitochondrial disorder that has lacked…
Professor Zdeněk Sofer of the University of Prague and his team developed and validated a "Lab-on-a-Scalpel" concept which is a surgical tool incorporating an integrated diagnostic sensor. Their findings were…
As noted in a recent article published in Inside Precision Medicine, in 1992 gene therapy was often discussed but seldom practiced. When Jim Wilson MD, PhD attended Penn State, gene…
Cowden Syndrome. A name that sounds almost gentle, until you realize it carries generations of uncertainty. It’s caused by changes in the PTEN gene and can affect the skin, thyroid,…
In a groundbreaking moment for transplant medicine, Massachusetts General Hospital (MGH) has concluded its second successful xenotransplantation of a genetically modified pig kidney into a human patient. Tim Andrews, a…
Portal Therapeutics, in partnership with GondolaBio, has achieved a milestone with the FDA granting orphan drug and fast track status to its oral therapy candidate, PORT-77. This investigational drug, according…
A groundbreaking study from the MRC London Institute of Medical Sciences (LMS) is reshaping how researchers approach treatment development for rare genetic disorders. Led by Dr. André Brown and the…
Sometimes our greatest lessons come from the moments that change us forever. Healing has not happened all at once for me. It has been a lifelong journey that began with…
Illumina, Inc. has unveiled its groundbreaking 5-base solution, setting a new standard for multiomic research by enabling scientists to simultaneously analyze genomic and epigenomic information from a single sample. According…
A recently published study in The Lancet reports that over 54,600 children living in Gaza are severely malnourished. In addition, as of August 2025, there are few therapeutic options available…
Merck has announced a significant expansion of its clinical development program for tulisokibart (MK-7240), an investigational monoclonal antibody targeting the inflammatory mediator TL1A. Reported by Drugs.com, in October 2025, the…
If you live in the Northern Hemisphere, Fall has quickly passed and Winter is upon us. The beginning of Fall is one of favorite times of the year with the…
© 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.
