In honor of World Duchenne Awareness Day on September 7th, Mal Duffin has invited us to share this app designed for parents whose children are living with Duchenne Muscular Dystrophy. Mal's…
The FDA has Completed Its Investigation of the Recent 2024 Deaths of Two Young Boys Due to Liver Failure. Acute failure of the liver has been found to be one…
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…
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 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…
Sarepta Therapeutics has announced a significant regulatory milestone in its efforts to secure permanent FDA approval for two breakthrough Duchenne muscular dystrophy (DMD) treatments. As reported by BusinesWire.com, the company…
The biotechnology sector experienced significant positive developments this week, with Xenon Pharmaceuticals' seizure medication achieving a decisive late-stage victory while regulatory pathways accelerated for several other promising therapies. According to…
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…
The biotech sector witnessed significant momentum as four clinical-stage companies announced compelling data across muscular dystrophy, immunology, and obesity treatment areas, triggering substantial stock market gains and investor enthusiasm for…
I’ve spoken regularly with family members and friends about my sons and their diagnosis and about our real-world struggles. I’ve advocated for them at our local pediatrician’s office, educated our…
ITF Therapeutics has announced the publication of encouraging long-term data supporting the efficacy and safety of givinostat as a treatment for Duchenne muscular dystrophy (DMD), according to a recent article…
Acknowledgement: This article was shared with Patient Worthy by our friends at Stork Genetics. To see the original article, please click here. For more information about women affected by X-linked…
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.…
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…
© Copyright 2024 Patient Worthy
Sign Up With a Patient Worthy Account and Share Your Rare Story
- OR -
Make a difference, share your experiences and get paid. Opt-in and join Patient Worthy's panel for paid opportunities such as surveys, market research, patient advisory panels and more.
- OR -
Make a difference, share your experiences and get paid. Opt-in and join Patient Worthy's panel for paid opportunities such as surveys, market research, patient advisory panels and more.
