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…
Editor's Note: Patient Worthy is proud to share this from our friends at the Courageous Parents Network. Throughout our lives we are faced with decisions of many kinds. Some involve…
Emerging research highlights the role of genetic predisposition in the development of secondary cardiomyopathies triggered by environmental stressors such as pregnancy, alcohol use, and cancer therapy. Findings published in JAMA…
Editor's Note: We're proud to be the first to share this story by Blair Young Whitworth, a Parent Champion of the Courageous Parents Network. There were so many anxious years…
Coffee lovers have long celebrated their favorite beverage for its taste and energy boost, but recent research offers another compelling reason to savor that daily cup: coffee directly and substantially…
Clinical trials often begin with optimism, sites are launched, projections look promising, and timelines are set. But when enrollment stalls, sponsors frequently call-in patient recruitment partners as a last-minute “rescue”…
Autoimmune diseases affect an estimated 400 to 600 million people globally, posing a growing challenge for healthcare systems and patients alike. Traditionally, treatment has focused on symptom management through broad…
As 2025 winds down, the pharmaceutical industry is bracing for a series of pivotal FDA decisions that could redefine treatment paradigms and reshape competitive dynamics across several major therapeutic areas.…
In a quiet corner of Texas, Jenna Hornbuckle is living a story so medically rare, doctors estimate its probability in the trillionth range. A single mother of two, Jenna is…
Editor's Note: Patient Worthy is honored to share this article from our friends at the Fatty Liver Foundation. We get a lot of questions about Tylenol and pain relievers in…
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