When Tucker Krause was a young boy, his family noticed that he frequently got sick. More than frequently, even. In fact, when his mom really thinks about it, Tucker was…
Don't forget to read Part 1 of our interview. In Part 1, we discussed what chronic granulomatous disease (CGD) is, how it is treated, and how parents and physicians can identify an…
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…
In a recent press release, clinical-stage gene therapy company Rocket Pharmaceuticals, Inc. ("Rocket") shared that its investigational gene therapy candidate, RP-L201, received Priority Medicines (PRIME) designation from the European…
A recent study published in the journal Blood Advances has documented that hematopoietic stem cell transplantation (HSCT) is both safe and effective for children with many different kinds of inherited nonmalignant conditions…
A recent publication from Specialty Pharmacy Times noted that the rapidly-growing number of available human immunoglobulin treatments for primary immunodeficiency disorders could pose challenges for specialty pharmacists when treating patients.…
According to a press release from New Jersey-headquartered ADMA Biologics, the FDA has approved the company’s primary humoral immunodeficiency disease (PIDD) drug Asceniv for the American market. The company expects…
Non-tuberculous mycobacterial (NTM) infections are caused by bacteria found in the environment. In healthy individuals, these bacteria normally cause no harm. However, for those who have a compromised immune system or who…
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…
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…
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;…
Conoce a Amanda! vlog de Amanda (video blog), ShAMANDAgans, le ayuda a mantenerse en contacto con amigos y familiares y compartir su historia. Ella es un ambicioso y alegre estudiante…
Imagine, if you will, that you've just been diagnosed with common variable immune deficiency, or CVID. The doctor has answered your most basic questions, and now you're on your way…
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…
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…
Amanda and Emily Gale are twin sisters who have never let primary immunodeficiency diseases (PI) stop them from achieving at the highest levels--literally. As reported on the Immune Deficiency Foundation's…
In case you didn’t know, October 11-17, 2015 marks the third anniversary of commemorating International Plasma Awareness Week. Sure, you might think that it’s just another ho-hum “awareness week” but…
Meet Amanda! Amanda’s vlog (video blog), ShAMANDAgans, helps her keep in touch with friends and family and share her story. She’s an ambitious and cheerful graduate student; going to…
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