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…
A child's life was recently saved thanks to ultra-ultra-rapid genome sequencing which provided him his rare disease diagnosis in just 13 hours. The Human Genome Project began 30 years ago…
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…
People are rushing to get in shape for summer; it's common to see the gyms a little more full than normal. While exercise is good for you, it's important not…
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…
As seen in a press release by Bioindustry; the UK BioIndustry Association (BIA) has published findings from a survey on the public’s interest in supporting rare access to affordable medicine…
A two-month-old baby recently traveled to Dubai to receive treatment at Al Zahra Hospital Dubai (AZHD), according to an article in the Khaleej Times. The child, who is from Oman,…
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