
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

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

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

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

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
Awareness starts with a conversation and action starts with screening. During Breast Cancer Awareness Month, let's encourage one another to learn about available resources, stay informed about screening recommendations, and prioritize preventive care. Together, we can help raise awareness, support those impacted, and promote the importance of early detection. 🎀 #BreastCancerAwareness #EarlyDetectionSavesLives #BreastCancerAwarenessMonth #PatientWorthy #h4tg #runforthehills ... See MoreSee Less
Today, we raise awareness for Phelan-McDermid syndrome and celebrate the individuals and families navigating life with this rare genetic condition. 💚💙 Every story matters, every individual deserves to be seen, and every step toward awareness brings us closer to greater understanding, inclusion, and support. Together, we can make a difference!
#phelanmcdermidsyndrome #phelanmcdermidsyndromeawareness #PatientWorthy
... See MoreSee Less

Hypertrophic cardiomyopathy (HCM) can affect each person differently. Symptoms may include shortness of breath, chest pain, or even fainting, while some people may have no symptoms at all. Knowing the symptoms and causes can help you understand if you're at a higher risk of diagnosis.
Have you or someone you love been affected by HCM? Click the link below to share your story.
ow.ly/G7qB50ZZl1q #hypertrophiccardiomyopathy #HeartHealth #PatientWorthy
... See MoreSee Less


+3
© Copyright 2024 Patient Worthy
Sign Up With a Patient Worthy Account and Share Your Rare Story
- OR -
Make a difference, share your experiences and get paid. Opt-in and join Patient Worthy's panel for paid opportunities such as surveys, market research, patient advisory panels and more.
What best describes you when it comes to rare disease? (check all that apply)
- OR -
Make a difference, share your experiences and get paid. Opt-in and join Patient Worthy's panel for paid opportunities such as surveys, market research, patient advisory panels and more.