According to Dr. Deepak Chellapandian, a Johns Hopkins specialist in bone marrow transplant for rare diseases, the prognosis for his patient was very poor. Jocelyn was born with chronic…
As reported in Biospace, the biotech company Jasper Therapeutics Inc. has announced the start of a Phase 1/2 clinical trial on a potential conditioning agent, JSP191, to facilitate hematopoietic cell transplants,…
According to a story from newshealth.biz, the results of recent research have revealed that recent use of acetaminophen can trigger a false-positive result on the dihydrorhodamine (DHR) test. This test…
Chronic granulomatous disease (CGD) is a rare form of immunodeficiency. The most common form of this disease is X-linked chronic granulomatous disease (X-CGD) which is caused by a mutated CYBB gene. This…
Orchard Therapeutics is investigating their gene therapy, OTL-102, for the treatment of X-linked chronic granulomatous disease (X-CGD). This treatment has recently received the Orphan Drug designation from the FDA, which…
According to a story from Newswise, a team of scientists and researchers associated with Université de Montréal and CHU Sainte-Justine have recently completed a study that maps a cellular model…
In a story from Healio, Dr. Niraj C. Patel, who is an associate professor of pediatric infectious disease and immunology at Levine Children’s Hospital, Atrium Health in Charlotte, NC, is…
According to a story from Markets Insider, the biopharmaceutical company Orchard Therapeutics recently announced the presentation of proof-of-concept data in regards to their investigational drug candidate OTL-102. This experimental therapy…
It may not be surprising news to find that the United States now pays more for prescription drugs than any other high-income country. This was not always the case however. Read…
When I was pregnant with my second child, I knew it would be different. We had all the essentials from having my daughter a few years earlier, and advice from…
Rare disease patients remain one of the most underserved medical populations, and they often have to deal with debilitating symptoms or complications that can make daily life difficult. For example,…
There’s been a lot of talk in recent years about interdisciplinary projects. The idea is to put together people with different skillsets to address problems. The goal is for everyone…
There is a long-standing debate on the ethical ramifications of testing medical procedures and drug treatments on animals. One side argues that nothing can replace the valuable information gathered from…
We've all heard it before: "Stay positive! Look on the bright side! Don't let the little things get you down!" The fact of the matter is it's way easier to…
Chronic granulomatous disease (CGD) is an inherited primary immunodeficiency disease associated with granulomas, which are clumps of immune cells that form at the site of infection or inflammation. As a…
Most kids don’t like to share. The occasional hand-me-downs? Sure. For me, my books, games, and beloved Dale Earnhardt hair bows were sacred property—mine, mine, mine. I chose not to…
If you’re lucky enough to find a job that you’re passionate about, it’s easy to see “work” as anything but. Still, love your job or not, it’s important to know…
Hey, do you guys remember that Mark Wahlberg movie that came out not long ago? You know, the one where he’s a cop? No no, not that one. The serious…
© Copyright 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.
