Editor’s Choice: Duchenne, GSD and Orphan Drugs
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…
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…