On July 21, 2021, Patient Worthy attended an online webinar presentation titled "How RDCA-DAP Can Help Inform Optimal Trial Design in Progressive Rare Disease." Organized by the Critical Path Institute…
The primary cause of death in melanoma patients is widespread metastases. Currently, there is no cure in cases of relapse or in advanced stages. A recent article in Science…
Have you ever heard of Bachmann-Bupp Syndrome (BABS)? If not, don't worry, you're not alone. In fact, BABS was only first discovered over the past few years. According to Medical…
The name of the organization is Disease InfoSearch. It is not only a resource for credible information but guides patients and families to valuable support groups and participation in…
This question is what the doctors at Massachusetts’ Cambridge Hospital were trying to determine. According to a recent article in the New York Times, a twenty-two-year-old man was rushed to…
Community support can be extremely uplifting and important, especially when one is impacted by a rare disease. This is the case for three-year-old Charlie Fry, who was born with SLC6a1.…
Dr. Jerry Vockley from the UPMC Children's Hospital of Pittsburgh has been studying the rare disease VLCAD for years. His work has led to the creation of two therapies which…
Compassion [kuhm-pash-uhn] noun A feeling of deep sympathy and sorrow for another who is stricken by misfortune, accompanied by a strong desire to alleviate the suffering. Compassion Corner is…
According to an article in NCBI’s Drug In Context, it took the combined services of the internal medicine, neurology, and psychiatry departments to diagnose this rare disease. Anti-NMDAR encephalitis…
Accessing treatments can be difficult, especially if one is impacted by a rare disease. In fact, only 5% of rare diseases have an FDA-approved treatment. Because of this, many patients…
Researchers at NYU Abu Dhabi (NYUAD) have discovered the code that is associated with the liver’s genome (complete set of DNA) and its ability to regenerate. According to a…
As reported in PR NewsWire; a pivotal Phase II/III trial to create a therapy for activated PI3K delta syndrome (APDS) has announced they’ve successfully completed patient enrollment for a new…
Welcome to Study of the Week from Patient Worthy. In this segment, we select a study we posted about from the previous week that we think is of particular interest…
ANGEL AID CARES began as the brainchild of Cristol Barrett O’Loughlin, out of a shared desire to pay tribute to her own family experience and to uplift others in need.…
The answer lies in the loss of endoplasmic reticulum (ER) calcium and protein which leads to cardiovascular, musculoskeletal, viral, and neurodegenerative diseases. ER performs many important cellular functions including…
As reported in I News; scientists may have found a cure for a devastating and fatal brain disease, and the world of rare brain diseases may never be the same.…
My nickname is Arti and I'm 11 years old. Because of my HAE diagnosis, I've been suffering from frequent and painful swellings since I was little. My life is divided…
Rare diseases face many obstacles when it comes to research and drug development. Small patient populations, a lack of awareness, and limited funding are just a few examples. When it…
Clustered Regularly Interspaced Short Palindromic Repeat (CRISPR), according to NCBI, are sequences that replicate and infect bacterial cells. CRISPR has been in the news recently, but this time it has…
According to a recent article in Genetic Engineering and Biotechnology News, the blood-brain barrier (BBB) regulates the movement of molecules, cells, and ions between the brain and the blood. Tightly…
Auto-Brewery Syndrome Auto-brewery syndrome is also known as gut fermentation syndrome. It occurs when fungi or bacteria within the gut produce ethanol by endogenous fermentation. Several different strains may be…
Newborn screening is important to early diagnosis and intervention. With some conditions, every second counts. Catching a rare disease as soon as a child is born could lead to much…
Patient Worthy was a media partner at this year's NORD Living Rare, Living Stronger Patient and Family Forum. This is an annual event hosted by the National Organization for Rare…
It's summertime, and that means tick season is upon us. We've all had to deal with the annoying insects, whether it's pulling them off our pets, spraying bug spray before…
We're learning more and more about COVID-19 every day. One thing that we're discovering as the pandemic continues is the complications that may arise after recovery. Chronic fatigue syndrome, brain…
© 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.
- 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.
