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…
Earlier this year, the FDA granted Accelerated Approval to ELEVIDYS (delandistrogene moxeparvovec-rokl) for the treatmnt of Duchenne muscular dystrophy (DMD) in boys aged 4-5. While this is a stunning…
In a story from BioPharma Dive, a number of high-profile clinical trials are under way that could see conclusive results by the end of 2023. While some of these are…
It looked as if the fate of Sarepta Therapeutics’ gene therapy for Duchenne muscular dystrophy was pretty much sealed until an FDA official intervened. This information was provided by…
Rare Community Profiles Rare Community Profiles is a new Patient Worthy article series of long-form interviews featuring various stakeholders in the rare disease community, such as patients, their…
Fierce Biotech recently carried an article about the death of Terry Horgan. Terry was a 27-year-old Duchenne muscular dystrophy (DMD) patient and the brother of the CEO of the…
As reported in Yahoo! Finance, the United States’ Food and Drug Administration (FDA) recently granted Orphan Drug designation to MP1032. This therapy, developed by clinical-stage biotech company MetrioPharm, is being…
Gene therapy has seemed out of reach for decades. Yet advocates have considered it a way to have damaged or missing genes restore dystrophin and other proteins. Doctors and advocates…
Rare Community Profiles Rare Community Profiles is a new Patient Worthy article series of long-form interviews featuring various stakeholders in the rare disease community, such as patients, their…
The citizens of Cincinnati know one universal truth: that the annual Flying Pig Marathon weekend will bring fun, family, and fulfillment to their city. And with that universal truth comes…
According to a story from PR Newswire, the biopharmaceutical company Avidity Biosciences, Inc., recently announced that its investigational therapy AOC 1044 has received the US Food and Drug Administration's Fast…
Medical research is incredibly important, especially within the rare disease community. Rare conditions tend to be under-researched and under-funded; this can make it difficult not only to better learn…
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