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.…
Heather White Heather White is a 41-year-old woman who was diagnosed with common variable immune deficiency (CVID) back in 2004 after fighting frequent bouts of pneumonia. Doctors believe that she…
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…
A young girl in Mauritius, an island in the Indian Ocean, has recently been given a second chance at a normal life after receiving a rare disease diagnosis and…
Vogmask is a San Francisco, California based company founded in 2011. They create stylish facial masks to protect wearers from inhalation of particulate and allergens. These types of masks can…
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…
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,…
Primary immunodeficiency diseases account for hundreds of rare and chronic diseases. In children, this may mean that their immune system is non-existent or doesn't perform as expected. To learn more…
Olivia Ports recently performed for over a thousand people, including the Mayor and Governor of Chicago, helping to raise $3.75 million for research by Lurie Children’s Hospital in only one…
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