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…
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…
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…
The European Commission has just granted their approval to Pharming for an extended indication of RUCONEST (conestat alfa) for Hereditary Angioedema (HAE). This extension allows the therapy to be utilized for…
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…
CSL Behring has recently received the Orphan Drug designation for Privigen for the treatment of systemic scleroderma. Privigen has also been granted the Fast Track designation. This therapy has been…
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…
The FDA has just provided Orphan Drug Designation to Hizentra for the treatment of adults diagnosed with chronic inflammatory demyelinating polyneuropathy (CIPD). This is a maintenance therapy which works to…
By Rachel Whetstone from In The Cloud Copy For millions of Americans who are affected by rare diseases, hope and health lies in the availability of plasma donations. One use…
With over 500,000 people affected by primary immunodeficiencies in the United States alone, experts are looking for ways to help doctors manage the needs of these patients. One significant…
WHIM Syndrome WHIM syndrome is a form of primary immunodeficiency. It is caused by a mutation in the CXCR4 gene. WHIM stands for Warts, Hypogammaglobulinemia, Infections, and Myelokathexis. Throughout their…
A New Study A recent study published in Nature Immunology and conducted by researchers at the University of Basel has produced influential results regarding primary immunodeficiency disorders (PID). These rare disorders…
According to a story from Cape Town Etc, two girls in South Africa were living with the same rare disease: primary immunodeficiency (PI), a group of rare conditions in which…
According to a story from peoplemagazine.co.za, Kasandra van Alphen is an eight year old girl from Johannesburg, South Africa that was diagnosed with primary immunodeficiency. Known by her friends and…
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 publication from BioSpace, New Jersey-based Kedrion Biopharma recently announced the enrollment of the first participant in their CARES10 study. CARES10 is a phase 3 study of a…
According to a story from The Indian Express, early treatment and diagnosis of primary immunodeficiencies makes management of this group of disease much simpler as a whole. In children, the…
According to a story from home.suddenlink.net, the biopharmaceutical company X4 Pharmaceuticals recently announced that the European Commission has granted Orphan Drug designation to the company's lead investigational drug candidate, mavorixafor.…
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