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Hunter Syndrome

Jay’s Hunter Syndrome Story

All genetic testing we did for Jay during pregnancy came back normal (though we did the bare minimum). When he was born, he struggled to

C. Diff

Experimental Treatment Targets C. diff While Sparing More of the Gut’s Microbes

A C. diff infection can turn a hospital stay into a much harder experience. The bacterium can cause severe diarrhea, abdominal pain and inflammation of

Parkinson's Disease

Claudine’s Deep-Dive into Parkinson’s Disease and Functional Neurologic Disorder

So, Parkinson’s Disease (PD) vs Functional Neurologic Disorder (FND) seem to join the other medical acronyms. What do these medical acronyms stand for? We have

Inclusion Body Myositis

A Journey of Hope, Strength, and Advocacy: Living with Inclusion Body Myositis

My journey with inclusion body myositis (IBM) has taught me that even when life takes an unexpected turn, hope, faith, determination, and the support of

Renal Cell Carcinoma

FDA Approves Belzutifan Plus Lenvatinib for Previously Treated Advanced Clear Cell Renal Cell Carcinoma

As reported on PharmaBiz, the US Food and Drug Administration (FDA) has approved the oral combination of belzutifan (Welireg) and lenvatinib (Lenvima) for adults with

Metabolic dysfunction-associated steatohepatitis

Late-Night Snacking, Sugar, and the Road to Liver Health

Many people spend very little time contemplating their liver, often ignoring this critical organ until a major medical issue arises. In contrast, health culture in

Lymphoma

When Cancer Comes With a Mortgage: How One Florida Town Turned a Beach Concert Into a Lifeline

Editor’s Note: This story was submitted to Patient Worthy by Roger Templeman, Executive Director of Stabilize Revitalize Fort Pierce (SRFP), a grassroots nonprofit in Fort

IgA Nephropathy

Transforming the IgA Nephropathy Treatment Landscape

For decades, patients diagnosed with IgA nephropathy—an autoimmune condition where trapped immune complexes inflame and damage the kidney’s filtering units (glomeruli)—faced a discouraging outlook. Typically

Metabolic dysfunction-associated steatohepatitis

AstraZeneca Expands AI-Driven Drug Discovery Efforts in MASH and Cardiomyopathy

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

Alexander disease

A New Chapter for Alexander Disease

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

Acute lymphocytic leukemia

My Sister’s Cells

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

Parkinson's Disease

Mission Therapeutics Begins Phase 1b Study of Experimental Parkinson’s Treatment

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

Hypertrophic Cardiomyopathy

Bristol Myers Squibb Wins FDA Expansion for Camzyos in Pediatric Obstructive Hypertrophic Cardiomyopathy

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),

Ebola

Ebola – Lessons Learned

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

CAPS Syndrome: A Summary, New Research, and Patient Stories

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.

Hunter Syndrome

Liam’s Hunter Syndrome Story

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

Primary Biliary Cholangitis

Colangitis biliar primaria: un diagnóstico que tarda demasiado

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)

Primary Biliary Cholangitis

Primary Biliary Cholangitis: The Diagnosis That Arrives Years Too Late

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