As reported on BioSpace, AstraZeneca has entered a major artificial intelligence-focused research collaboration with biotechnology company insitro, committing up to $1.5 billion in potential payments
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
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
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
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),
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
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.
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
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)
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
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
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
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
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
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
A Chicago cancer conference this summer revealed an astounding development – a recent clinical trial of a new therapy, Daraxonrasib, was nearly doubling survival rates
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
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
Indousrare🎤 We’re Honoured to Welcome Karim Mikhail, Director of the Center for Biologics Evaluation and Research (CBER), FDA, as Keynote Speaker at the 2026 Bridging RARE Gala 💙
Bringing new treatments to patients takes years of research, collaboration, and careful evaluation. With his experience across biopharma and regulatory leadership, Karim brings an important perspective to the ongoing efforts to advance rare disease care.
We look forward to welcoming Karim and others working to advance rare disease care for an evening dedicated to recognizing meaningful contributions, building connections, and working towards a better future for people living with rare diseases.
📅 November 1, 2026 | 5:00–10:00 PM
📍 Hylton Performing Arts Center, Manassas, Virginia
🌐 Learn more: www.indousrare.org/gala/
🎟️ Register for the Gala: bit.ly/3TGryiM
#BridgingRARE #RareDisease #RareDiseaseAwareness #RareDiseaseCommunity #IndoUSrare
@Pitthopkins
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