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;…
Contributed by Jane Larkindale and Alayna Tress While millions of people globally are living with a rare disease, patients often find it difficult to feel seen or heard throughout their…
The current standards-of-care for those with Duchenne muscular dystrophy (DMD) include corticosteroids, immunosuppressive treatments, physical therapy, and occupational therapy (among others). Gene therapy has the potential to change the treatment…
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…
Before you read on, don't forget to check out Part 1 of our interview with Jennifer Wallace Valdes, PT. In Part 1, Jennifer discusses her background, what Duchenne muscular dystrophy (DMD) is,…
Occupational therapy for individuals with Duchenne muscular dystrophy (DMD) requires a unique and specialized approach to maximize benefit and reduce harm. It is incredibly important that those performing occupational therapy…
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…
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.…
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…
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…
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.…
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…
Sometimes, it can be difficult to spur drug development for rare or life-threatening diseases, considering the number of patients. To address this, and improve patient outcomes, the FDA developed the…
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…
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