UK Moves to Make Whole Genome Sequencing Standard in the NHS, Improving Diagnosis for Rare Disease Patients

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…

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RUCONEST Now Approved for Children with Hereditary Angioedema in Europe

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…

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New Method of Infusion for Primary Immunodeficiency Could Improve Patients; Quality of Life
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New Method of Infusion for Primary Immunodeficiency Could Improve Patients; Quality of Life

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…

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April 22nd Kicks of World Primary Immunodeficiency Week 2020
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April 22nd Kicks of World Primary Immunodeficiency Week 2020

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…

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What if the World Always Thought of the Immunocompromised as They are Right Now?
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What if the World Always Thought of the Immunocompromised as They are Right Now?

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…

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ICYMI: Privigen Granted Orphan Drug Designation For Treatment of Systemic Scleroderma
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ICYMI: Privigen Granted Orphan Drug Designation For Treatment of Systemic Scleroderma

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…

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ADMA Biologics Earns BioNJ 2020 Innovator Award for Primary Immunodeficiency Treatment
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ADMA Biologics Earns BioNJ 2020 Innovator Award for Primary Immunodeficiency Treatment

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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Orphan Drug Designation Provided to a Treatment for Chronic Inflammatory Demyelinating Polyneuropathy
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Orphan Drug Designation Provided to a Treatment for Chronic Inflammatory Demyelinating Polyneuropathy

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…

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The Current Plasma Shortage is Worrying to Those Who Depend on Treatments

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…

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The Importance of Patient-Centered Care Primary Immunodeficiencies
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The Importance of Patient-Centered Care Primary Immunodeficiencies

  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…

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Breakthrough Therapy Designation Given to Investigative WHIM Syndrome Treatment

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…

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PID Patients Could Receive More Personalized Treatment Thanks to New Study

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…

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Two Best Friends With Primary Immunodeficiency Meet for the First Time in Cape Town, South Africa
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Two Best Friends With Primary Immunodeficiency Meet for the First Time in Cape Town, South Africa

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…

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Specialty Pharmacists Play Important Role in Treatment of Primary Immunodeficiency Patients
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Specialty Pharmacists Play Important Role in Treatment of Primary Immunodeficiency Patients

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.…

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Enrollment Opens for Phase 3 Study of Intravenous Immunoglobulin in Primary Immunodeficiency 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…

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Primary Immunodeficiency in Children: Early Intervention is Important
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Primary Immunodeficiency in Children: Early Intervention is Important

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…

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Possible Treatment for WHIM Syndrome Earns Orphan Drug Designation in the EU
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Possible Treatment for WHIM Syndrome Earns Orphan Drug Designation in the EU

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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A Plasma Shortage is Affecting Common Variable Immune Deficiency Patient’s Access to Treatment

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…

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Teenage Girl Walks Again After Finally Getting Diagnosed With Complement C1q Deficiency and Bone Marrow Transplant
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Teenage Girl Walks Again After Finally Getting Diagnosed With Complement C1q Deficiency and Bone Marrow Transplant

  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…

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Vogmask Offers a Stylish Face Mask for Those Living with Immunodeficiencies

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…

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X4 Pharmaceuticals Reports Positive Phase 2 Results for WHIM Syndrome Drug

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…

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A New Study Aims to Improve Diagnoses for People with WHIM Syndrome
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A New Study Aims to Improve Diagnoses for People with WHIM Syndrome

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,…

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No-Cost Disease Diagnosis and Immunoglobulin Therapies Now Available to Children with Primary Immunodeficiency Diseases
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No-Cost Disease Diagnosis and Immunoglobulin Therapies Now Available to Children with Primary Immunodeficiency Diseases

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…

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