Lebanese Family Travels Across the World to Find Treatment for Child with SCID
What could make a family in Lebanon travel all the way to UCLA? The answer is a unique stem cell gene therapy designed to combat SCID. The El Kerdi family…
What could make a family in Lebanon travel all the way to UCLA? The answer is a unique stem cell gene therapy designed to combat SCID. The El Kerdi family…
What could make a family in Lebanon travel all the way to UCLA? The answer is a unique stem cell gene therapy designed to combat SCID. The El Kerdi family…
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…
Researchers from St. Jude Children's Hospital might have just developed a new gene therapy that can build a healthy, functioning immune system for patients suffering from severe combined immunodeficiency, SCID,…
'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…
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…
Remember when PW reported on the Newborn Screening and Gene therapy for the Immune Deficiency SCID? Well guess what our friends at the Immune Deficiency Foundation are doing? The IDF…
Doctor, por favor revise la sangre de mi bebé! La IDF alienta a los funcionarios estatales del estado de Louisiana a firmar en rutinas de detección de recién nacidos…
La terapia genética se ha hablado desde hace bastante tiempo, pero las noticias recientes han convertido la esperanza en realidad para los bebés nacidos con adenosina desaminasa (ADA). Los bebés…
Cuando me uní a otros millones de fans obsesionados que veían lo que ha sido la popular episodio "Bubble Boy" en Seinfeld en 1992, no tenía ni idea de que…
It takes an average of seven years for a patient with a rare disease like severe combined immunodeficiency (SCID) to receive a correct diagnosis; and that comes after the patient…
Happy Fall Friday Patient Worthians! This week we have the amazing story about a woman with dystonia. We also have a video review of a man diagnosed with CVID. Additionally,…
Happy Friday Patient Worthians! We have two intriguing stories from PW contributors this week, detailing their plight with rare disease. Additionally, we have a pretty cool outcome for a pretty cool kid…
Imagine you're a new parent and your two-month-old starts experiencing some bizarre symptoms. Along with his high fever, his skin turns red and starts peeling at an alarming rate. Multiple infections follow;…
You may have seen the Jeffery Modell Foundation's name around town, literally, from their educational posters. I was thrilled to come across one in the Chicago O'Hare International Airport. The…
Severe combined immunodeficiency (SCID)—one of the many forms of primary immune deficiencies. You may also know it as the "Bubble Boy" disease. Here at Patient Worthy, we've talked a…
One of the many problems with having a rare disease is that very few people know anything about what you, or your loved one, go through on a day-to-day basis.…
You may have heard the buzz words "gene therapy" without really understanding what it is, or what's at stake. Gene therapy is when a healthy copy of a gene replaces…
At PatientWorthy, we've talked a lot about gene therapy, but we've never really taken the time to explain it. What the heck is gene therapy? How does it work? Well, like…
A couple of months ago we took a look at the legacy of David Vetter, the “Boy in the Plastic Bubble” whose lifelong battle with Severe Combined Immunodeficiency (SCID) caught…
Gene therapy has been talked about for quite some time, but recent news has turned a hope into a reality for babies born with adenosine deaminase (ADA)-deficient SCID, severe combined…
Doctor, please take my baby's blood! The IDF encourages Louisiana state officials to sign into law routine screenings of newborns for SCID, or Severe Combined Immune Deficiency. The IDF's ultimate goal is to…
When I joined millions of other obsessed fans who watched what’s become the wildly popular “Bubble Boy” episode on Seinfeld back in 1992, I had no idea there was a shred…