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
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
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
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
One death is too many – Especially when it can be prevented. The new MGB policy will benefit patients who are at risk of deadly
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
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
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
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
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
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
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 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
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.
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
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,
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
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
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