Happy Friday! This week we have details about an investigative therapy for Duchenne Muscular Dystrophy that has entered a phase 3 clinical trial. Also, have you ever heard of Glycogen…
Oftentimes, the rare disease journey can be difficult. Diagnosis may take years. Some conditions are still relatively unknown by doctors. Families must not only learn about these conditions, but how…
Many Twitter users were confused and concerned when Julie O'Mahony posted a picture of her mother's hand with two of the fingers white and completely drained of color. However, while…
On April 28th, the virtual World Orphan Drug Congress USA 2021 was held. The program featured a variety of subjects relevant to the development of orphan drugs and the rare…
Did you know that an estimated 30% of patients with Parkinson's disease (PD) experience diplopia (double vision)? According to Parkinson's News Today, a large longitudinal study determined that diplopia is…
For many people, fish oil supplements are part of their health routine. I know that I have a bottle sitting in my vitamin cabinet. These supplements offer omega-3 fatty acids…
Chiprez Ramirez describes her younger brother Francisco as her "little ray of sunshine." Talking to MSN, she said that his rare disease journey and strength are what motivate her to…
Welcome to the Rare Classroom, a new series from Patient Worthy. Rare Classroom is designed for the curious reader who wants to get informed on some of the rarest, most…
The National Kidney Foundation has partnered with Bayer to launch and run the "Are You the 33%?" campaign, which intends to raise awareness about the heightened risk of chronic kidney…
In a recent news release, pharmaceutical company Tarsier Pharma ("Tarsier") shared that it held a successful pre-Investigational New Drug (pre-IND) meeting with the FDA. The meeting focused on TRS02, a…
Most cancers are inflamed (hot tumors) and under the right conditions can be controlled by T-cells. According to a recent article in Genetic Engineering and Biology News, there is another…
According to a story from apnews.com, the biopharmaceutical company FibroGen, Inc., has recently announced that its investigational treatment pamrevlumab has earned Rare Pediatric Disease designation from the US Food and…
TG Therapeutics has been developing ublituximab as a treatment for chronic lymphocytic leukemia (CLL) when used in combination with umbralisib, marketed as UKONIQ. The biopharmaceutical company has recently completed and…
According to a story from Benzinga, there are at least eight drug companies that are currently working on a treatment for Rett syndrome, a rare genetic disorder. With no cure…
According to MarketScreener, patients with endometrial cancer in Europe now have a new treatment option. Recently, the European Commission (EC) granted conditional marketing authorization to GlaxoSmithKline for their therapy JEMPERLI…
According to a recent news release, CTX001, a gene-edited therapy for patients with transfusion-dependent beta thalassemia, received Priority Medicines (PRIME) designation from the European Medicines Agency (EMA). The treatment was…
Is it or was it COVID? That is the question doctors and parents are asking as the number of children admitted to the hospital for a rare pediatric inflammatory…
Jaxson Corcoran was born with tuberous sclerosis complex (TSC), a rare genetic disorder that is characterized by benign tumors forming throughout the body. Treating this condition has become a family…
British researchers recently conducted a study of 235,379 COVID-19 survivors over a six-month period after they survived their initial infection. The researchers discovered that one out of three patients…
As Prevention reminds us, tick season is officially upon us! That's right, these little pests enjoy warm and humid weather. So even while we're still social-distancing, ticks are getting ready to show…
According to a story from the Knowridge Science Report, a recent study conducted by the Japanese Kumamoto University indicates that a therapy commonly used to treat diabetes, called metformin, can…
In the last decade, scientists have made great strides in uncovering details of the underlying causes of neurodegenerative diseases. It is only a matter of time that treatment and prevention…
Tim Revell has run the Ascension Seton Austin Marathon a whopping 15 times, with his 16th run planned for this Sunday. The father of two runs the race for his…
My name is Adam Nelson. I am 33 years old and I was born with a rare genetic connective tissue disorder known as epidermolysis bullosa simplex (EBS) of the Weber-Cockayne…
Welcome to the Rare Classroom, a new series from Patient Worthy. Rare Classroom is designed for the curious reader who wants to get informed on some of the rarest, most…
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.
