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…
In 2020, Chris and Susan Finazzo received news that would change the trajectory of their lives. They learned that their sons Chase (now 9) and Dylan (now 6) had a…
When Jason Fox was just a child, doctors told his family that he would most likely not live to 18 years old. He had been diagnosed with Duchenne muscular dystrophy…
The Griffins of Harrisburg, North Carolina enrolled their son in the AGAMREE clinical trial in 2016. Their son was the first participant in the trial studying the investigational drug vamorolone…
For over 20 years, CureDuchenne, a Patient Worthy partner and global nonprofit, has moved steadfastly forward with a mission to find, and fund, a cure for individuals with Duchenne muscular…
Sarepta Therapeutics’ treatment Elevidys (delandistrogene moxeparvovec-rokl) received conditional approval from the FDA in June 2023. The designation implies that if the drug is used in accordance with its label, it…
CureDuchenne, a global leader in research and patient care for Duchenne muscular dystrophy (DMD), has been working to find and fund a cure for DMD for more than two decades.…
The research landscape for Duchenne muscular dystrophy (DMD) has been steadily increasing. Researchers are exploring more therapies, including gene therapies, that could transform the lives of those with this rare…
Vamorolone, now marketed as AGAMREE®, is termed a dissociative steroid therapy, as it has proven its potential to retain the muscle-strengthening and anti-inflammatory benefits of corticosteroids. On the other side…
Rare Community Profiles 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,…
For over four decades, the aspiration of gene therapy has been to develop novel therapies with the potential to enhance human health. Recombinant AAV (rAAV), a type of gene therapy,…
Since its founding 20 years ago, CureDuchenne has been tirelessly working to find and fund a cure for Duchenne muscular dystrophy (DMD). They have done this through supporting families,…
NS Pharma, Inc., a subsidiary of Nippon Shinyaku Co., recently announced via news release that its therapy NS-089/NCNP-02 was granted Orphan Drug designation from the U.S. Food and Drug…
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