What is primary immunodeficiency? The Mayo Clinic offers this definition: "PI weakens the immune system and as a result infections or other health problems may occur more often. People with…
Welcome to the Rare Classroom, a new series from Patient Worthy. Rare Classroom is designed for the curious reader who wants to get informed on some of the rarest, most…
In the past, treatment for WHIM syndrome has focused on symptom management: treating infections and myelokathexis with antibiotics or granulocyte colony stimulating factor. Infusion can treat hypogammaglobulinemia and surgery…
In a late July 2023 news release, global healthcare company Grifols shared that positive topline data was now available from a Phase 4 clinical study evaluating XEMBIFY (immune globulin…
A primary immunodeficiency (PI), or primary immune deficiency disease, is an inherited immune system disorder caused by gene mutations which prevent the immune system from functioning properly. It can…
The coronavirus pandemic has significantly altered not just the world we live in, but the way we engage within this new world. It has been difficult and isolating to…
When Branden was just a young child, he was diagnosed with a rare genetic disorder called chronic granulomatous disease (CGD). Now, at 33 years old, Branden is passionate about raising…
Genetic testing has always been costly and directed at DNA sequencing. Only a limited number of genes could be tested. It was useful to a small number of patients…
The COVID era emphasized the importance of maintaining a strong immune system. But what about people who are less fortunate? Johns Hopkins researchers recently announced some good news for…
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…
A research study led by Professor Beishi Zheng from Columbia University has studied the immunogenetics of gastrointestinal (GI) cancers. The Study This study demonstrated that individuals who are diagnosed with…
X4 Pharmaceuticals Inc. has recently published data from 3 different posters which were presented at the American Society of Hematology (ASH) Annual Meeting in Georgia. You can read the full…
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…
It is extremely important to get the right medications into the hands of patients who need them. Recently, the FDA helped do this for pediatric patients (aged 2+) with primary…
Underserved communities have difficulties when trying to access the resources that are available to the majority. Because of this, more work has to be done to ensure that everyone can…
About a month ago, from June 23-26, The Immune Deficiency Foundation hosted the 2021 Primary Immunodeficiency Conference. The event was the largest gathering of primary immunodeficiency (PI) patients across the…
According to a July 13 news release, biopharmaceutical company Kedrion Biopharma ("Kedrion") recently treated its first pediatric patient within the Phase 3 KIDCARES10 clinical trial. Within the trial, Kedrion…
The Primary Immunodeficiency Conference June 23-26, 2021 This conference is the largest primary immunodeficiency (PI) patient gathering on Earth. Patients, clinicians, and caregivers will come together for this virtual event…
Welcome to the Rare Classroom, a new series from Patient Worthy. Rare Classroom is designed for the curious reader who wants to get informed on some of the rarest, most…
According to a recent press release, biopharmaceutical company Kedrion Biopharma ("Kedrion") achieved an important milestone in its Phase 3 CARES10 trial: the last treated patient. During the CARES10 trial, researchers evaluated…
A recent study has demonstrated that through standardizing the whole genome sequencing process and making it routine for patients, we can improve rare disease diagnosis across the board. Earlier diagnosis…
A New Study A new study, conducted by CLS Behring, has found that a new method of administration for Hizentra as a treatment for primary immunodeficiency (PI), could improve quality…
April 22nd, 2020 heralds the beginning of World Primary Immunodeficiency (PI) Week. This will be the ten year anniversary of the event, which is designed to spread awareness about PI…
If the world were as careful in everyday life Perhaps I’d be sick less, I’d feel much less strife It's the small precautions, like washing your hands That for me…
According to a story from gurufocus.com, the biopharmaceutical company ADMA Biologics, Inc., has earned the 2020 BioNJ Innovator Award. This award was given as acknowledgement of the company's successful development…
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