As reported on BioSpace, the U.S. Food and Drug Administration (FDA) has granted priority review to BridgeBio Pharma’s investigational therapy, BBP-418, marking a key regulatory milestone for a condition that…
As reported on PharmaBiz, Regenxbio has announced encouraging topline findings from the pivotal Phase III portion of its ongoing AFFINITY DUCHENNE clinical program evaluating RGX-202, an investigational gene therapy for…
Brussels — On February 24, 2026, the acclaimed Netflix documentary The Remarkable Life of Ibelin received the Media & Awareness Raising Award at the EURORDIS Black Pearl Awards. The honor…
NS Pharma, Inc., a subsidiary of Nippon Shinyaku Co., Ltd., has received a significant regulatory boost for its experimental treatment targeting Duchenne muscular dystrophy (DMD). As reported in The AI…
Our youngest son, Jace, is 11 years old with Duchenne Muscular Dystrophy. It is a fatal muscle-wasting disease, and there is currently no cure. We were given the diagnosis on…
A recent story featured in The Sun highlights the life-changing potential of a newly approved drug for Duchenne muscular dystrophy (DMD), as seen through the eyes of a young boy…
Not everyone who stands up for children’s health in Washington, DC, wears a suit or carries a briefcase. Sometimes, the most powerful advocates are the ones who have experienced the…
The FDA recently informed Muscular Dystrophy News that it has granted orphan drug designation to the experimental drug ISX9-CPC, a product of the IPS Heart company located in Houston, Texas.…
Individuals with Duchenne muscular dystrophy have mutations in the gene that makes the protein dystrophin. This gene is the largest gene in the human body, with 79 exons. When there…
According to a recent article, 17-year-old Diego Ramirez was able to compete in the Flying Pig Marathon despite his Duchenne muscular dystrophy diagnosis. Duchenne Muscular Dystrophy (DMD) Duchenne muscular dystrophy…
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! November 1 is International Acromegaly Day. For those unaware, Acromegaly is a rare condition characterized by the presence of an elevated level of growth hormone and typically effects…
Happy Friday! Monday, Sept. 7 is Duchenne Muscular Dystrophy Awareness Day. DMD is characterized by the inability to make the protein dystrophin in muscles and can result in heart and…
As originally discussed in Muscular Dystrophy News Today, a recent study has discovered that increasing levels of utrophin in the body can improve muscle strength, form, and function for patients…
Scientists are researching the possibility of fetal gene therapies as possible cures for rare diseases, such as Duchenne muscular dystrophy, hemophilia, sickle cell disease, and Gaucher's disease. With fetal gene…
Duchenne muscular dystrophy (DMD) is a heartbreaker for the the 250,000 families worldwide whose sons are born with this debilitating, progressive disease. It is rare, and yet the ripples of its…
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