According to an article published by Biospace, X4 Pharmaceuticals, based in Cambridge, Massachusetts, has recently announced successful phase two results in clinical trials for their experimental drug X4P-001. The drug…
According to a story from BioSpace, the biotechnology company X4 Pharmaceuticals recently announced that the US Food and Drug Administration (FDA) has given Orphan Drug designation to the company's investigational…
Just days after my CVID diagnosis, I was invited to a support dinner. It was at this support dinner that I became aware of how unique my situation was; I…
Researchers are exploring a new diagnostic method that could be applied to newborn screening for certain inherited diseases and monitoring of HIV patients. For more detailed information, you can click…
A gene therapy treatment is being used to treat X-linked severe combined immunodeficiency (X-SCID). Mustang Bio has licensed the treatment from St. Jude Children's Research Hospital. X-SCID is also known…
Clinical data from a Phase 2 study of the experimental drug X4P-001-RD for the treatment of WHIM syndrome has been announced at the European Hematology Association’s 23rdCongress in Stockholm. The…
A primary immunodeficiency report, published in the journal Clinical and Experimental Immunology, examines medical care for primary immunodeficiency patients in the UK. The publication of the report coincides with the…
A large clinical study will be carried out to develop a protocol for identifying a rare primary immunodeficiency disease called WHIM syndrome in patients, reports BioPortfolio. The Jeffrey Modell Foundation,…
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,…
'Tis the season for gene therapy approvals. Reported by Bloomberg, the U.K. just approved its most expensive medication to date. The treatment is for severe combine immune deficiency, or more…
Joy Littlesunday is just one of many Navajo newborns who were screened and thus saved from severe combined immunodeficiency by a successful bone marrow transplant at UC San Francisco, reported UCSF…
As seen by her story on IDF's website, for Kayla Kuehl, her diagnosis of an extremely rare disease happened over the course of several years. It's called Common Variable Immune Deficiency…
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…
Scratchy jeans. No. 2 pencils. Trapper Binders. Squeaky shoes. Floor polish. Helpful bus drivers. Smiling teachers. Ah, the scents, sights and sounds of the first day of school. I loved…
No one likes being sick, yet Mom always finds a way to turn this rough situation into a special feeling. Mothers have a way of taking care of you physically…
Primary immunodeficiency diseases (PI) are a group of more than 300 rare and chronic disorders where part of the body's immune system is missing or does not function properly. Since…
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…
When my son was born he didn't exhibit primary immunodeficiency (PI) or any of the other conditions on the newborn screening panel. Still, I fretted over just about everything. He…
Once upon a time there were three young brothers, and SPOILER ALERT- this part of the story, unfortunately, does not have a happy ending. Each brother suffered: Each had chronic…
Want some more news, events and announcements on Batten disease? We got 'em! [one_half] [/one_half] [one_half_last] Pioneering Brain Surgery May Give This Toddler More Time FDA Press Release…
The concept of a “library” is something we probably take for granted—but at its core, the library is one of humanity’s greatest innovations. The act of collecting information for the…
When you have an immunodeficiency disease, you’re very much at risk of getting an opportunistic infection because your immune system isn’t “scanning” your body’s cells to kill off abnormal or…
Primary Immunodeficiency (PI) can be very hard to explain to people and very hard to understand. The thing is, most of the time we look healthy on the outside. But…
Want some more news, events and announcements on SCID? We got 'em! [one_half] [/one_half] [one_half_last] The Expense of Scientific Progress: The Price of Gene Therapy for SCID The…
Chances are if you have a primary immunodeficiency disorder, you don’t live near other teens with your same diagnosis. No one else gets what you’re going through. You may have…
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.
