On September 3, 2025, the US Food and Drug Administration (FDA) introduced the Rare Disease Evidence Principles (RDEP) process, a groundbreaking step for drug development targeting ultra-rare genetic diseases. According…
Luke McCarthy of Killybegs is preparing to take on the Galway Bay Marathon, not just as a personal fitness goal, but as a heartfelt tribute to his father, Joe, who…
Editor's Note: Patient Worthy is proud to share this article from our friends at the TESS Research Foundation. To see the article in its original format, please click here. TL;DR:…
On Sunday, more than 100 medical experts from across the globe gathered in Rochester for the Mayo Clinic’s four-day “Undiagnosed Hackathon”—a pioneering event dedicated to solving rare diseases that have…
After years of determined advocacy and scientific perseverance, Stealth BioTherapeutics has achieved a landmark victory with the FDA’s accelerated approval of elamipretide (now branded as Forzinity) the first-ever treatment for…
Avacopan, a novel oral therapy for antineutrophil cytoplasmic antibody-associated vasculitis (ANCA-AAV), has changed treatment paradigms for this rare but serious disease. However, until now, a validated technique for measuring avacopan…
In our third installment from our series of reports provided by our partner TREND Community, we are bringing you several reports that analyze the effects of sleep disorders. This week…
An estimated 47% of infants have some form of plagiocephaly (a flat spot on the head), and up to 10% will eventually need a corrective helmet to help reshape their skull. Some…
In a breakthrough study, scientists have uncovered a surprising ally in the fight against cancer: the bacteria living within tumors. According to World Pharma News, an international team led by…
Editor's Note: Patient Worthy is proud to share this story from our friends at No Stomach for Cancer. To see the article in its original form, please click here. In…
Life has a way of catching us off guard when we least expect it. For me, it all started with severe stomach pains that led to an unexpected hospital visit.…
Research into rare diseases has long faced obstacles, from limited patient numbers to the challenge of replicating disease biology in the lab. A new wave of innovation, highlighted in the…
The U.S. Food and Drug Administration (FDA) has introduced a new regulatory framework aimed at improving and clarifying the approval process for drugs targeting rare diseases, also known as orphan…
Editor's Note: Patient Worthy is honored to share this article from our friends at Courageous Parents Network. Parents of well children regularly receive anticipatory guidance–proactive communication about what’s coming in…
Editor's Note: This article is an opinion piece shared with Patient Worthy from our partner network. Opinion pieces may not reflect the views of Patient Worthy, its employees, or any…
Ireland has unveiled a new national strategy aimed at transforming the diagnosis and care of the estimated 300,000 people in the country living with rare diseases. According to The Irish…
Families and researchers in North Carolina are making urgent appeals to the FDA, highlighting the desperate need for faster approval of drugs for rare and ultra-rare diseases that threaten children’s…
In our second installment from our series of reports provided by our partner TREND Community, we are going to be focusing on a different condition over four weeks. This week…
The U.S. Food and Drug Administration (FDA) is reviewing the first medication specifically developed for Barth syndrome, a rare and life-threatening genetic disorder. As reported by STAT, this medication could…
A recent report from NDTV World brings attention to a rare but significant public health event: an American man has tested positive for plague, the infectious disease historically known for…
Health authorities in Queensland, Australia, have issued a public warning after the discovery of Naegleria fowleri—a rare but deadly “brain-eating” amoeba—in a town’s water supply. As reported by The Medical…
A routine evening became pivotal for a Portsmouth family when a bright white reflection appeared in nine-month-old Nora’s left eye under bathroom lights. As was reported by Mirror, what seemed…
Acknowledgement: Patient Worthy is honored to share this story by Erika Stariha, Co-Founder and President of SATB2 Europe. This is Urban, my teenage firstborn. He is joyful. Curious. Full of…
In our third installment from our series of reports provided by our partner TREND Community, we are going to be focusing on a different condition over four weeks. This week…
After more than a decade of development, families say time is running out for children with Barth syndrome as the experimental therapy elamipretide faces yet another regulatory delay. Reported by…
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