As reported on BioSpace, Sarepta Therapeutics has reached an important regulatory milestone in its effort to secure traditional FDA approval for two Duchenne muscular dystrophy (DMD) treatments, Amondys 45 and…
Editor's Note: This story was submitted to us by Angelina Olivera, the mom of a 14-year-old with Duchenne muscular dystrophy, and the sister of two brothers who died from Duchenne…
As reported on BioSpace, the U.S. Food and Drug Administration (FDA) has granted priority review to BridgeBio Pharma’s investigational therapy, BBP-418, marking a key regulatory milestone for a condition that…
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…
Chugai Pharmaceutical Co., Ltd. announced that Roche has made the strategic decision to discontinue clinical development of GYM329 (emugrobart), an investigational anti-latent myostatin antibody, for two neuromuscular conditions: spinal muscular…
Editor's Note: This story was submitted to Patient Worthy by Ghulam Ali, Founder & CEO, Muscular Dystrophy Pakistan. I am 35 years old and hold a Bachelor’s degree in Rural…
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…
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…
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