Cancelleri’s son, Levi, was diagnosed with Alexander disease as a baby. Above, he smiles widely, wearing the shirt his dad made. Photo courtesy of Tom Cancelleri Editor's Note: Patient Worthy…
Editor's Note: This story was shared with Patient Worthy by our friends at Elephants & Tea. The article, originally written by Kimberly Oliver, can be found in its original format…
The international study will examine whether MTX325 affects processes linked to Parkinson’s disease, as well as assess its safety and tolerability. As reported in a recent press release, Mission Therapeutics says…
As reported on Yahoo! News, Bristol Myers Squibb (BMS) has received an expanded approval from the US Food and Drug Administration (FDA) for Camzyos (mavacamten), extending its use to pediatric…
The Ebola outbreak in DR Congo was declared in May 2026. As reported in BBC News, authorities in DR Congo recently confirmed that the virus is spreading at an alarming…
CAPS, or Cryopyrin-Associated Periodic Syndromes, is a group of rare autoinflammatory diseases caused by a genetic mutation affecting the cryopyrin protein, leading to increased inflammation. CAPS is usually linked to…
Editor's Note: This story was originally written by Anne Denneen, and shared on Caring Bridge. So many things happen in life that you don’t even register them at the time. …
Nota del editor: Para Patient Worthy es un honor compartir este artículo, que nos fue enviado originalmente por Andrés Contreras. La colangitis biliar primaria (PBC) es una enfermedad crónica del…
Editor's Note: Patient Worthy is honored to share this article, originally submitted to us by Andres Contreras. Primary biliary cholangitis (PBC) is a long-term liver disease in which the immune…
Editor's Note: Patient Worthy is honored to bring you this article, shared with us by our friends at Rareatives. Originally written by Davi Jacobs, the article can be found in…
Diagnosis and year of diagnosis I was diagnosed with epilepsy in 2000, and I have been living with epilepsy for more than 26 years. My journey has included countless seizures,…
Sixteen years ago, when my daughter was diagnosed with a rare autoimmune disease, I knew I was scared. I knew I was overwhelmed. I knew I was stressed. What I…
At just two years old, Asher is already teaching us that the smallest milestones can be the biggest victories. Our son, Asher, was born on February 10, 2024. He's a…
When there is no treatment for your child's rare disease, how do you find a way forward? For one mother in the Czech Republic, the answer was to start asking…
A Chicago cancer conference this summer revealed an astounding development – a recent clinical trial of a new therapy, Daraxonrasib, was nearly doubling survival rates for late-stage pancreatic cancer patients.…
The University of Michigan has opened a $10 million research center dedicated to histotripsy, a technique that uses focused sound waves to destroy tumors. The center will make specialized equipment…
Editor's Note: Patient Worthy is honored to share this story from our friends at Elephants & Tea. Originally written by Mellory Torres, a survivor of acute lymphocytic leukemia, the article…
Fabry disease is a rare inherited condition caused by changes in the GLA gene. These changes can lead to low or absent activity of an enzyme called alpha-galactosidase A. As…
Bangladesh did not meet its 2026 measles target due to vaccination gaps in 2024 and 2025 according to a report in Reuters. The following year saw cancellation of a nationwide…
IndoUSrare and GORD Foundation are honored to announce the 2026 Abbey Meyers Khushi Bridging RARE Award Honorees: Rare Disease is a global problem, a human problem, and the individuals below…
Editor's Note: Patient Worthy is honored to present this story, originally written by Blyth Lord, founder of Courageous Parents Network. I was asked earlier this week by a high school…
Nota del editor: Para Patient Worthy es un honor compartir este artículo, que nos fue enviado originalmente por Andrés Contreras. El cáncer de hígado es la tercera causa de muerte…
Editor's Note: Patient Worthy is honored to share this article, originally submitted to us by Andrés Contreras. Liver cancer is the third leading cause of cancer death worldwide. In the…
My name is Kelli Frederick, M.A., and I have lived with neurofibromatosis for my entire life. I have also undergone more than 50 facial surgeries. When you live with a…
Two papers published in Nature Communications report new findings about roginolisib, an experimental cancer drug being developed by iOnctura. One study describes how the drug interacts with a cancer-related enzyme…
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