Betty Vertin is a mother, writer and a fierce defender of the decisions she and her husband Jason made about the seven children they brought into this world. Betty writes…
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…
As reported on BioPharmaDive, Roche is restarting efforts to get Elevidys, a gene therapy for Duchenne muscular dystrophy (DMD), approved in Europe by launching a new global Phase 3 trial…
Sarepta Therapeutics has announced a significant regulatory milestone in its efforts to secure permanent FDA approval for two breakthrough Duchenne muscular dystrophy (DMD) treatments. As reported by BusinesWire.com, the company…
As reported on the Manila Times, Aucta Pharmaceuticals has officially introduced PYQUVI™ (deflazacort) oral suspension 22.75 mg/mL, marking the company’s entry into the U.S. commercial branded‑generic market. The therapy, released…
CureDuchenne is hosting several free events in 2026 to support families living with Duchenne and Becker muscular dystrophy. These events, called CureDuchenne CARES, will take place across the country. At…
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…
The biotech sector witnessed significant momentum as four clinical-stage companies announced compelling data across muscular dystrophy, immunology, and obesity treatment areas, triggering substantial stock market gains and investor enthusiasm for…
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…
I’ve spoken regularly with family members and friends about my sons and their diagnosis and about our real-world struggles. I’ve advocated for them at our local pediatrician’s office, educated our…
ITF Therapeutics has announced the publication of encouraging long-term data supporting the efficacy and safety of givinostat as a treatment for Duchenne muscular dystrophy (DMD), according to a recent article…
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…
In a pivotal development for the neuromuscular disease community, the European Commission has granted approval to a new therapy for Duchenne muscular dystrophy (DMD), marking a significant milestone in the…
Duchenne muscular dystrophy (DMD) is a devastating, inherited muscle-wasting disorder caused by mutations in the DMD gene, leading to an absence of functional dystrophin protein. While current standards of care,…
Duchenne Muscular Dystrophy (DMD) is a severe, progressive neuromuscular disease that primarily affects boys, leading to muscle weakness, loss of ambulation, and premature death. Among therapeutic advances, ataluren has emerged…
The Accredited Duchenne Centers (ADC) Program, spearheaded by the World Duchenne Organization (WDO), is a pioneering global initiative designed to standardize and elevate care for individuals living with Duchenne Muscular…
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…
Written by Chuck Howe Nothing in life gives me greater joy than seeing my grandchildren be happy and thrive. You can imagine the heartbreak and fear I experienced when I…
Did you know that World Duchenne Day is recognized each year on September 7th? This day is dedicated to spreading awareness about Duchenne muscular dystrophy (DMD), a rare disease. Patient…
Pfizer has recently paused its Phase III clinical trial that has been assessing the Duchenne muscular dystrophy candidate fordadistrogene movaparvovec. The Phase II DAYLIGHT trial (NCT05429372), enrolled ten boys ages…
Rare Community Profiles is a Patient Worthy article series of long-form interviews featuring various stakeholders in the rare disease community, such as patients, their families, advocates, scientists, and more.…
Duchenne muscular dystrophy (DMD) is an inherited disease that weakens a child’s muscles and eventually spreads throughout the child’s body. DMD is caused by mutations in the gene that helps…
The Duchenne muscular dystrophy (DMD) community has a reason to celebrate. According to a recent news release, the FDA recently approved Duvyzat (givinostat) for people ages six and older who…
Editor's Note: Chronic conditions and rare diseases don't discriminate, Patient Worthy and its partners are interested in amplifying the voices of those from all identities and backgrounds. If you have…
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