BridgeBio Pharma has reported decisive success in a pivotal phase 3 trial for its rare disease drug BBP-418, aimed at treating limb-girdle muscular dystrophy type 2I/R9 (LGMD2I/R9). Reported by Fierce…
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…
The FDA recently informed Muscular Dystrophy News that it has granted orphan drug designation to the experimental drug ISX9-CPC, a product of the IPS Heart company located in Houston, Texas.…
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…
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…
There are over 30 subtypes of congenital muscular dystrophy (CMD), or muscular dystrophies that are present at birth. These rare genetic disorders often manifest in hypotonia (low muscle tone) and…
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…
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…
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…
Prior to launching a clinical trial, the FDA must clear an Investigational New Drug (IND) application. This allows the drug to be distributed across state lines, which is important…
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…
Congenital muscular dystrophy (CMD) affected individuals, Kelly Berger and Avery Roberts are making their voices heard. Together they hope to tackle misconceptions surrounding disability and give a loud voice…
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