Happy Thursday! This week, we have details on the beginnings of a new trial testing CRISPR gene editing for Duchenne muscular dystrophy, how parents can play a critical role in…
September 7 is World Duchenne Day, a day to help spread awareness about Duchenne muscular dystrophy among the general public and in the medical field. In recognition of this day,…
Business Wire published an August 10th news release announcing that Cure Rare Disease, a non-profit based in Boston, Massachusetts received FDA approval to administer CRD-TMH-001 (CRD), its first-ever therapeutic.…
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…
The Orphan Drug designation was created to aid and hasten the development of drugs created for the treatment, diagnosis, or prevention of rare diseases. When the FDA grants this designation,…
As we know, many treatments for rare diseases are quite expensive - and this price doesn't even factor in travel, lodging, appointments, and more. To help with this, the government…
CureDuchenne is a nonprofit whose mission is to find a cure for Duchenne muscular dystrophy (DMD), a rare progressive disease characterized by muscular degeneration. It is diagnosed in one individual…
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…
On the first Sunday in May, Cincinnati, OH hosts the Flying Pig Marathon – its 26.2 mile race. This year marked the 24th annual event. But for 17-year-old Diego Ramirez,…
CureDuchenne is one of Patient Worthy's partner organizations. Patient Worthy partners with a variety of rare disease and patient-oriented non-profits in order to collaborate and help promote one another's activities.…
Betty Vertin recently wrote an article discussing her experience as a parent of a child with Duchenne muscular dystrophy. She shares the lessons she has learned in order to…
According to a recent article, a research team discovered that the inhibition of sphingolipid synthesis on mice models of Duchenne muscular dystrophy can counteract the certain symptoms of the disease.…
Our parents can be some of our strongest supporters, and Hawken Miller knows this firsthand. He recently published an article in Muscular Dystrophy News Today detailing the love and support…
According to a recent press release from Dyne Therapeutics, the FDA has placed a clinical hold on their Investigational New Drug (IND) application for DYNE-251. Until this hold is lifted,…
According to a November 23 article from Muscular Dystrophy News, the FDA recently granted Orphan Drug designation to clinical-stage biotechnology company Regenxbio's experimental gene therapy candidate, RGX-202. The gene therapy,…
Two and a half years after announcing its launch, Dyne Therapeutics held its very first Research & Development Day. Throughout the day, there were presentations focusing on the development of…
Funding for clinical trials can be an issue, especially for rare disease studies. Luckily, there are programs that exist to help with the financials, such as the "Clinical Studies of…
It's a beautiful thing when a community pulls together to support one of its members. This is exactly what the Madeira community is doing for 16-year-old Diego Ramirez. He recently…
According to a recent news item that appeared on The Denver Channel, we are now in the age of genetic research that could have an impact on the lives of…
September 7, 2021 will be recognized as World Duchenne Awareness Day. This day will be set aside in recognition of awareness surrounding Duchenne muscular dystrophy, a rare and debilitating genetic…
On July 21, 2021, Patient Worthy attended an online webinar presentation titled "How RDCA-DAP Can Help Inform Optimal Trial Design in Progressive Rare Disease." Organized by the Critical Path Institute…
Beijing, China: According to an article in a sister publication of Sixth Tone, a woman living in China who has been treated during the past fourteen years for paroxysmal nocturnal…
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…
According to Muscular Dystrophy News, new research suggests that the cholesterol metabolic pathway could offer a potential therapeutic target for patients with Duchenne muscular dystrophy (DMD). Following microRNA analysis, researchers…
Clinical trial data is extremely helpful in understanding the safety, efficacy, and tolerability of various potential treatments. According to Muscular Dystrophy News, top-line data from the Phase 2b VISION-DMD…
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