According to a story from MENAFN, the Delhi High Court recently issued a directive to the National Consortium for Research, Development, and Therapeutics for Rare Diseases to convene and move…
CureDuchenne, the nation’s leading nonprofit dedicated to funding a cure for Duchenne muscular dystrophy (DMD) and a Patient Worthy partner, held its first “Napa in Newport” event in 2015;…
According to an October 31, 2022 press release from muscle disease company Dyne Therapeutics, Inc. ("Dyne"), the company's therapeutic candidate DYNE-251 earned Fast Track designation from the FDA for Duchenne…
According to a story from Patient Worthy partner CureDuchenne, the organization has recently launched its CureDuchenne Occupational Therapist Certification Program. This program is intended to give occupational therapists advanced skills…
Chances are, you’ll never meet anyone like Lizanne, a 22-year-old woman from the Netherlands. Not only is she extraordinarily passionate about storytelling and filmmaking, but she was diagnosed with a…
In the past, myofibers (terminally differentiated post-mitotic cells that help make up muscle tissue) have been implicated in the development and progression of Duchenne muscular dystrophy (DMD). Basically, researchers have…
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.…
CureDuchenne Live Workshop August 6, 2022 Little Rock, AR A FULL DAY EVENT FOCUSED ON IMPROVING THE QUALITY OF LIFE FOR INDIVIDUALS WITH DUCHENNE. CureDuchenne Cares is an interactive education…
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,…
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…
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…
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…
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…
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