A U.S. House of Representatives oversight panel has sharply criticized the Health Resources and Services Administration (HRSA) following the release of a report revealing serious shortcomings in the country’s organ…
Ultragenyx Pharmaceutical, a biotechnology company developing treatments for rare diseases, has received a setback from the U.S. Food and Drug Administration (FDA) regarding its gene therapy candidate, DTX401. According to…
A significant step forward in the treatment of a rare and painful skin condition has emerged from Finland. According to News Medical Life Sciences, Tampere University has announced the licensing…
I live with a rare, genetic disease called familial chylomicronemia syndrome, or FCS, which prevents my body from breaking down fats and removing triglycerides from my bloodstream. FCS is inherited…
The study, published in the journal The Lancet Neurology and reported by MedicalXpress.com, is the first to demonstrate the urgency of extending imaging that includes the heart. This must be accomplished within…
Epidermolysis Bullosa (EB) is a devastating and rare genetic skin disorder often described as having "butterfly skin." For those living with EB, even the gentlest touch can lead to painful…
“I have to be dying”, I said earnestly to my husband for the second time that year, “at the very least, I don’t think there is much more pain I…
This article was originally published on Ask the Patient. Welcome to the inaugural post of “Ask the Patient.” As a cancer survivor who has experienced medical gaslighting, medical bias, negligent…
In North Texas, a remarkable young girl named Isabella "Izzy" Esquivel has found a unique way to experience happiness and self-expression—through her art. Izzy, now ten years old, was diagnosed…
A new potential treatment for light chain amyloidosis is making significant strides in clinical development. According to a recent update reported by Drugs.com, the phase III clinical program called “CARES”…
On July 18, 2025, the U.S. Food and Drug Administration (FDA) took decisive action against Sarepta Therapeutics following the deaths of three individuals who had received the company’s gene therapy…
Patient Worthy is honored to share Oliver's story on behalf of SynGAP Research Fund. SRF is a global group of families committed to accelerating the science to cure SYNGAP1 &…
One death is too many - Especially when it can be prevented. The new MGB policy will benefit patients who are at risk of deadly toxicities from a widely prescribed…
A major new study from Denmark has provided real-world evidence that HPV vaccination among girls leads to strong and lasting protection against the most dangerous HPV types responsible for cervical…
In a groundbreaking medical achievement, eight babies have been born in the UK free from incurable mitochondrial disease, thanks to a pioneering fertility technique using DNA from three people. The…
Instead of transplanting a heart that has been cooled and stopped, the new technique, at times referred to as “heart in a box”, creates a scenario where the heart that…
A recent story featured in The Sun highlights the life-changing potential of a newly approved drug for Duchenne muscular dystrophy (DMD), as seen through the eyes of a young boy…
Takeda Pharmaceuticals has announced positive results from a pivotal Phase 3 trial of oveporexton, a novel therapy targeting orexin deficiency in patients with narcolepsy. According to BioPharmaDive.com, this development marks…
A significant advancement for people living with hereditary angioedema (HAE) arrived with the recent FDA approval of Ekterly (sebetralstat), the first oral on-demand therapy designed to treat acute HAE attacks…
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…
A groundbreaking study has revealed that the balance of metabolites produced by gut microbes (measured from a simple stool sample) can predict the short-term risk of death for critically ill…
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,…
The concept of “food as medicine” has gained significant traction in recent years, and for good reason: mounting evidence shows that nutrition is a powerful tool in both the prevention…
At the recent American Society of Clinical Oncology (ASCO) annual meeting, researchers unveiled remarkable long-term results for ciltacabtagene autoleucel (cilta-cel; Carvykti), a CAR T-cell therapy, in patients with heavily pretreated…
A groundbreaking advance in diabetes care has emerged from recent clinical research: a new cell therapy has successfully restored natural insulin production in 83% of participants living with diabetes. This…
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