A new Nordic study has revealed a significant link between blood clots caused by paroxysmal nocturnal hemoglobinuria (PNH) and an increased risk of kidney failure. PNH is a rare, life-threatening…
The U.S. Medicaid program has taken a pioneering step to ensure that new gene therapies for sickle cell disease (SCD) are accessible to the millions of Americans who rely on…
Editor's Note: This story was originally authored by Natalie H., a cancer survivor and loving mother of two. They say cancer changes everything. I’ve learned it doesn’t just change you,…
Acknowledgement: Patient Worthy is honored to share this story from our friends at GACI Global. To view the original article, please click here. Sharon’s passion for children shines in her…
A groundbreaking study has demonstrated that gene therapy can successfully restore hearing in patients with hereditary deafness, offering new hope for individuals affected by genetic forms of hearing loss. The…
ScanCell, a biotech firm focused on immunotherapies, announced promising results from Phase 2 clinical trials for its skin cancer vaccines, but only one of its two candidates will advance to…
A growing focus on patient-reported outcomes (PROs) is transforming the management of Paroxysmal Nocturnal Hemoglobinuria (PNH) and aplastic anemia, two rare and serious blood disorders. According to a recent article…
Patient Worthy is grateful to provide you this story on behalf of the European Society for Phenylketonuria and Allied Disorders Treated as Phenylketonuria. E.S.PKU is the umbrella organization of about 41…
A new investigational therapy targeting neutrophils, a type of white blood cell, may offer a safer and more effective option for patients with ulcerative colitis (UC), according to recent research…
The global market for treating Paroxysmal Nocturnal Hemoglobinuria (PNH), a rare but serious blood disorder, is poised for significant growth through 2034, according to a recent analysis by DelveInsight reported…
Danielle’s baby boy had a long and terrifying seizure on the 4th of July six years ago. The family was at her mother’s home. Both Danielle and her mother are…
A new study has found that dogs are capable of detecting Parkinson’s disease by scent, potentially opening the door to earlier and less invasive diagnosis for the neurological disorder. According…
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…
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