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.…
FUTURES National Conference May 23-26, 2024 Orlando, FL The CureDuchenne FUTURES National Conference is an annual event focused on bringing education, connection, and hope to the entire Duchenne community. Get…
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…
CureDuchenne Cares Dinner Session April 13, 2024 Pittsburgh, PA A NEIGHBORHOOD DINNER FOR CAREGIVERS AFFECTED BY DUCHENNE AND BECKER JOIN US FOR A CASUAL THREE-HOUR DINNER ON SATURDAY EVENING TO…
CureDuchenne Cares Workshop April 6, 2024 Long Island, NY A FULL DAY EVENT FOCUSED ON IMPROVING THE QUALITY OF LIFE FOR INDIVIDUALS WITH DUCHENNE AND BECKER. CureDuchenne Cares is an…
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…
CureDuchenne Cares Dinner Session March 16, 2024 Tampa, FL A NEIGHBORHOOD DINNER FOR CAREGIVERS AFFECTED BY DUCHENNE AND BECKER JOIN US FOR A CASUAL THREE-HOUR DINNER ON SATURDAY EVENING TO…
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…
Champions in Miami 2024 March 9, 2024 Miami, FL This gala is not just an exquisite gathering; it serves as a significant source of support for CureDuchenne, empowering the organization…
CureDuchenne Cares Workshop March 9, 2024 Baltimore, MD A FULL DAY EVENT FOCUSED ON IMPROVING THE QUALITY OF LIFE FOR INDIVIDUALS WITH DUCHENNE AND BECKER. CureDuchenne Cares is an interactive…
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…
CureDuchenne Cares Dinner Session February 24, 2024 Charlotte, NC A NEIGHBORHOOD DINNER FOR CAREGIVERS AFFECTED BY DUCHENNE AND BECKER JOIN US FOR A CASUAL THREE-HOUR DINNER ON SATURDAY EVENING TO…
CureDuchenne Cares Workshop February 10, 2024 Atlanta, GA A FULL DAY EVENT FOCUSED ON IMPROVING THE QUALITY OF LIFE FOR INDIVIDUALS WITH DUCHENNE AND BECKER. CureDuchenne Cares is an interactive…
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,…
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