Patient Worthy's award-winning podcast "Wait, How Do You Spell That? A Rare Disease Podcast" is back with a new episode. This week, Colby is sitting down with Valen Keefer, a…
Researchers at Washington University’s medical school have used a new technique to study brain neurons in a lab setting, allowing them to bypass the need to rely on a brain…
Rare Community Profiles is a Patient Worthy article series of long-form interviews featuring various stakeholders in the rare disease community, such as patients, their families, advocates, scientists, and more.…
The U.S. Food and Drug Administration has announced a public meeting set for Wednesday, October 16, 2024 to discuss the creation of a Rare Disease Innovation Hub. The meeting is…
By: Erin Santos-Primis, Mom to Isabella and Executive Director of the Isabella Santos Foundation Starting a nonprofit in honor of a loved one lost to a tragic disease is often…
Rare Community Profiles is a Patient Worthy article series of long-form interviews featuring various stakeholders in the rare disease community, such as patients, their families, advocates, scientists, and more. Jonathan…
First established by the International Foundation for Gastrointestinal Disorders (IFFGD) in 2016, Gastroparesis Awareness Month is designed to increase awareness of gastroparesis and how it affects the people who have…
After years of research an international team of scientists has unraveled the genetic makeup of 47 strains of known and potential Lyme disease-causing bacteria. The work paves the way toward…
Fecal microbiota transplants (FMTs) take healthy bacteria from stool provided healthy individuals, and transfer it to a donor with gut microbiome dysbiosis. Typically, fecal transplants are performed to treat C.…
Globally, an estimated 39.9 million people live with human immunodeficiency virus (HIV). HIV is an infection which, over time, damages the immune system. This condition can be spread through sexual…
At Japan’s Fujita University, researchers reported to Inside Precision Medicine that a new risk measurement may add a layer of accuracy in predicting sudden cardiac death. The measure, called estimated…
As I reflect on over 20 years living with dystonia and pain and other symptoms that changed my life in many ways, I want to share some of the things…
In some cases, dermatomyositis can be difficult to treat. A variety of treatment options are available, from corticosteroids and immunosuppressants to antimalarials, colchicine, alendronate, and warfarin. But this rare condition…
Have you ever heard of cytokines? These signaling proteins play a role in inflammation in your body. Normally, cytokines help your immune system respond to foreign invaders. But when too many…
By: Harsha K Rajasimha, Ph.D., Founder and CEO of Jeeva Clinical Trials Challenges Unique to Cell and Gene Therapies (CGTx) Clinical Trials Cell and gene therapies (CGTx) have emerged as…
The month of August is recognized as Spinal Muscular Atrophy (SMA) Awareness Month. This is a time of year in which the community works to spread awareness about this rare…
Rare disease drug development can be challenging. Countries globally developed programs to incentivize innovation and development in this space. One of these initiatives is the designation of orphan drugs, or…
On July 11th Dr. Mathew Winton went to Capitol Hill with senators and staff from both parties to discuss collaboration with the FDA regarding the pending termination of the Priority…
Patient Worthy's award-winning podcast "Wait, How Do You Spell That? A Rare Disease Podcast" is back with a new episode. This week, Colby is sitting down with professional speaker and…
The long-awaited positive results of Annexon Inc.’s Phase 3 trial investigating ANX005 for Guillain-Barré syndrome (GBS) was presented at the PNS 2024 Annual Meeting. Several leading global GBS experts spoke…
Ruby Carr has always loved playing outside. But an enjoyable day in the summer sun turned into chaos after Ruby was unknowingly bitten by a tick. As the tick latched…
In unedited, early-access research published in Nature, a team of researchers explain that an estimated 60% of people with neurodevelopmental disorders remain undiagnosed, despite comprehensive genetic testing options. In many cases, this is…
Yusara Ahmed was prepared to take action when she learned that her son Yusuf was diagnosed with beta thalassemia, an uncommon blood disorder. Yusara's sister also lived with the disease,…
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