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…
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…
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…
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…
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…
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…
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…
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…
© 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.
