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…
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…
June 2025 marked a month of significant progress in cancer care, as the FDA approved several new therapies and treatment combinations across a range of cancer types. As reported by…
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…
For decades exercise has been recommended to improve the quality of life. More recently and perhaps more importantly, exercise has been highly recommended for cancer patients. Most people realize that…
KalVista Pharmaceuticals has achieved a significant milestone with the U.S. Food and Drug Administration’s (FDA) approval of its new oral therapy for hereditary angioedema (HAE), marking a breakthrough for patients…
A new milestone has been reached in the search for effective treatments for dry age-related macular degeneration (AMD), as the first patient has now been dosed in a clinical trial…
Fujifilm Corporation’s investigational therapy FF-10832 has received Orphan Drug Designation from the U.S. Food and Drug Administration (FDA) for the treatment of biliary tract cancer, according to a recent announcement…
Editor's Note: This article was originally published on 3/5/25, and is being republished due to ongoing efforts by Patient Worthy to raise awareness of COPD. A presenter at the COPD…
My name is Kelly Baughman, and I grew up in a small town in Louisiana—tight-knit, faith-driven, and yes, I went to church and summer camp with the Duck Dynasty family.…
The United States is experiencing its worst measles outbreak in decades, with cases rising sharply across multiple states. Health officials attribute this surge primarily to declining childhood vaccination rates, fueled…
A recent study has brought attention to the complex challenges faced by children with hidradenitis suppurativa (HS), a chronic and often painful skin condition. According to new research summarized by…
A new study has found that corticosteroids, commonly used to manage symptoms in lung cancer patients, may reduce the effectiveness of immunotherapy treatments. The research, highlighted by News-Medical.net, sheds light…
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