Fast Track designation is granted by the FDA to facilitate the development and expedite the review of drugs to both fill unmet needs and treat serious or life-threatening conditions. Through…
During this year’s commemoration of Rare Diseases, Leonard Saig M.D. a director at Bnei Zion Haifa spoke about Cushing’s syndrome, also called hypercortisolism. Cushing's syndrome is rare and difficult to…
Rare diseases can impact many aspects of life, whether that is physical, psychological, educational, or something else. As caregivers are some of the closest people to those with rare diseases,…
In late February 2022, biopharmaceutical company Trascenta Holding Limited ("Trascenta") provided an update on its clinical program for TST001, a monoclonal antibody therapy designed for those with locally advanced or…
It's likely that you've heard of meningitis before - it occurs when the membranes around the brain and spinal cord become inflamed. A number of sources can lead to this…
UCB Inc., a biopharmaceutical company, has announced positive results for its RAISE Phase 3 trial (NCT04115293) investigating zilucoplan against placebo. According to a recent article in Biospace, zilucoplan met…
Want to learn about scientific topics without needing a PhD? Check out the Science Simplified blog from TESS Research Foundation! Dr. Tanya Brown, PhD, works with researchers to make science…
Welcome to Study of the Week from Patient Worthy. In this segment, we select a study we posted about from the previous week that we think is of particular interest…
The following story was told to R29 Fertility Diaries by a 34-year-old mother and her husband. The newly married couple was anxious to be parents. Even after the loss of…
When Kacey Lamphier was just a child, her father Edward passed away from a ruptured aorta. Following his death, Kacey and her family were also tested. It was during this…
In January of this year, a University of Connecticut (UConn) team administered the world’s first infusion of a new gene therapy as part of a long-awaited clinical trial. According to…
The war on opioids inadvertently became a war on severely ill patients. The "war" had to be declared. There were reactions from all fronts to the realization that deaths rose…
One thing that we know about psoriatic disease (a term that refers to both psoriatic arthritis and psoriasis) is that is linked to a heightened risk of cardiovascular (CV) mortality…
Because there are so many rare diseases that we still have so much to learn more about, there are many instances where we believe that diseases overlap or are variations…
The Australian Sports Brain Bank was established in 2018 with a goal of better understanding chronic traumatic encephalopathy (CTE) and other conditions associated with repeated head injuries or blows to…
This Saturday, March 12, marks Narcolepsy Awareness Day, also known as Suddenly Sleepy Saturday. The Narcolepsy Network aims to raise awareness and urges everyone to do their part to advocate…
Patient Worthy is a partner of Personalize My Medicine, which is dedicated to a patient-focused approach to medical innovation and research. PMM can help you organize a personalized approach to…
Note: This is part two of a two part story. Don't forget to go back and read part one! Meghan O’Rouke, the author of the NYT best-selling book Invisible Kingdom,…
When Emily McGuyre first discovered that she was pregnant, she was thrilled. She greatly looked forward to bringing her son, Matthew, into her family. But, shares The Hawkeye, Emily learned…
This is an interview I did with Dr. Carly Hudson on the Healing Ground Movement podcast discussing my life with dystonia and chronic pain, as well as the various ways…
According to a story from Biopharma Dive, the US Food and Drug Administration (FDA) has recently approved a treatment for a rare form of melanoma that affects the eyes, called…
A recent study conducted at the Penn State College of Medicine focused in on nonalcoholic fatty liver disease (NAFLD), specifically blood clotting in this rare disorder. These researchers knew that…
In the past, you’ve probably heard about chronic traumatic encephalopathy (CTE) in relation to football. This rare neurodegenerative disorder has been linked to repeated blows to the head. But did…
Myrtelle, via Business Wire News, reports the completion of Phase 1/2 investigating its gene therapy for Canavan disease (CD) which they have now expanded to treat younger patients. Thus far,…
Biotechnology company Genmab A/S recently shared that its investigational therapy epcoritamab (DuoBody-CD3xCD20), being developed with AbbVie, was granted Orphan Drug designation from the FDA. The therapy is designed for those…
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