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…
As reported by drugs.com, at the American College of Rheumatology (ACR) Convergence congress, Novartis presented encouraging results from its Phase III NEPTUNUS-1 and NEPTUNUS-2 trials, highlighting the potential of ianalumab…
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…
On September 3, 2025, the US Food and Drug Administration (FDA) introduced the Rare Disease Evidence Principles (RDEP) process, a groundbreaking step for drug development targeting ultra-rare genetic diseases. According…
Editor's Note: Patient Worthy is proud to share this article from our friends at the TESS Research Foundation. To see the article in its original format, please click here. TL;DR:…
On Sunday, more than 100 medical experts from across the globe gathered in Rochester for the Mayo Clinic’s four-day “Undiagnosed Hackathon”—a pioneering event dedicated to solving rare diseases that have…
Avacopan, a novel oral therapy for antineutrophil cytoplasmic antibody-associated vasculitis (ANCA-AAV), has changed treatment paradigms for this rare but serious disease. However, until now, a validated technique for measuring avacopan…
The U.S. Food and Drug Administration (FDA) has introduced a new regulatory framework aimed at improving and clarifying the approval process for drugs targeting rare diseases, also known as orphan…
Families and researchers in North Carolina are making urgent appeals to the FDA, highlighting the desperate need for faster approval of drugs for rare and ultra-rare diseases that threaten children’s…
The U.S. Medicaid program has taken a pioneering step to ensure that new gene therapies for sickle cell disease (SCD) are accessible to the millions of Americans who rely on…
A recent research article in ScienceDirect.com details a breakthrough in visualizing the rapid, complex movements of proteins using high-speed atomic force microscopy (HS-AFM). This cutting-edge imaging technology allows scientists to…
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…
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…
Collaboration has always made the world go round - and it's no different in rare disease research. Sometimes, the community affected by a certain rare disease is small, which…
Jörg Richter is no stranger to difficult bike rides. In fact, shares KRDO, Richter has ridden his bike across the United States - from coast to coast - seven times…
Don't forget to check out Part 1 of our interview with Katheron Intson, where we discussed her background, her research experience in GRIN1, and the reasons behind why she developed…
Nancy Yu co-founded AllStripes with a vision in mind: to unlock more treatment options for those with rare diseases. In Part 1 of our interview, Patient Worthy sat down with…
Clinical trials face many obstacles regardless of which disease they aim to investigate. However, rare disease trials face additional problems that don't typically impact studies for more common conditions. Small…
Rare diseases often need extra support when it comes to research and drug development. Luckily, MMS Holdings Inc. (MMS) plans to provide some with their new support program. The program,…
Rare disease research usually isn’t the first thing med students think to specialize in. Mostly, because it’s such a small field and it’s not extensively covered during medical school. Let's…
On May 12th, 2017 the University of Rochester Medical Center hosted the first Technology and Rare Neurological Diseases Symposium (TRNDS). It successfully brought together patients, researchers, leaders in health technologies,…
One of the first things many people do after being diagnosed with a rare disease is search online to find out more about it. I know that is exactly what…
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