Study Has Led to Better Understanding on the Difference Between Activated PI3K Delta Syndrome 1 and 2
source: pixabay.com

Study Has Led to Better Understanding on the Difference Between Activated PI3K Delta Syndrome 1 and 2

APDS Activated PI3K delta syndrome (APDS) is a form of primary immunodeficiency (PI). There are fewer than 100 reported cases of the condition. APDS has two types (1 and 2),…

Continue Reading Study Has Led to Better Understanding on the Difference Between Activated PI3K Delta Syndrome 1 and 2
April 22nd Kicks of World Primary Immunodeficiency Week 2020
source: pixabay.com

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…

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

Review of Rare Movement Disorders in India and the United States

A recent article published in India’s Pharma Literati cites The World Health Organization as not having endorsed a definition for rare diseases. The European Union defines 'rare' as less than…

Continue Reading Review of Rare Movement Disorders in India and the United States
What if the World Always Thought of the Immunocompromised as They are Right Now?
source: pixabay.com

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…

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

Building Community Among Children with Rare Diseases: The Fighting H.A.R.D. Foundation

The Fighting HARD Foundation Rare diseases are difficult to face for a myriad of reasons. They can be financially burdensome, physically painful, and emotionally draining. Further, they can make patients…

Continue Reading Building Community Among Children with Rare Diseases: The Fighting H.A.R.D. Foundation
Scientists Find That Rubella and Certain Cancers Have the Same Mutation Mechanisms
source: pixabay.com

Scientists Find That Rubella and Certain Cancers Have the Same Mutation Mechanisms

By Danielle Bradshaw from In The Cloud Copy Scientific teams at the Centers for Disease Control (CDC), the National Institute of Environmental Health Sciences (NIEHS), and other research centers have…

Continue Reading Scientists Find That Rubella and Certain Cancers Have the Same Mutation Mechanisms

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…

Continue Reading The Current Plasma Shortage is Worrying to Those Who Depend on Treatments
The Current Plasma Shortage is Worrying to Those Who Depend on Treatments
source: pixabay.com

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

For millions of Americans who are affected by rare diseases, hope and health lies in the availability of plasma donations. One use of the donated plasma involves the extraction of…

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

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…

Continue Reading Breakthrough Therapy Designation Given to Investigative WHIM Syndrome Treatment
More than 4% of People May be Immunocompromised – How Should They Approach Flu Season?
source: pixabay.com

More than 4% of People May be Immunocompromised – How Should They Approach Flu Season?

A recent publication from Healio detailed some of the challenges immunocompromised individuals might face during flu season. Influenza is a common and sometimes serious public health concern around the world, and…

Continue Reading More than 4% of People May be Immunocompromised – How Should They Approach Flu Season?
Five Month Old Patient in the Spotlight for Primary Immunodeficiency Walk
stanvpetersen / Pixabay

Five Month Old Patient in the Spotlight for Primary Immunodeficiency Walk

According to a story from the Warwick Beacon, the first ever primary immunodeficiency (PI) awareness and fundraising walk in Rhode Island's history was held recently at Warwick City Park. The…

Continue Reading Five Month Old Patient in the Spotlight for Primary Immunodeficiency Walk
Primary Immunodeficiency in Children: Early Intervention is Important
RachelBostwick / Pixabay

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…

Continue Reading Primary Immunodeficiency in Children: Early Intervention is Important
Company Starts up Late Stage Trial Testing Experimental WHIM Syndrome Treatment
mwooten / Pixabay

Company Starts up Late Stage Trial Testing Experimental WHIM Syndrome Treatment

According to a story from BioPortfolio, the biopharmaceutical company X4 Pharmaceuticals, Inc. has recently initiated a phase 3 clinical trial that is testing its current lead product candidate mavorixafor. This…

Continue Reading Company Starts up Late Stage Trial Testing Experimental WHIM Syndrome Treatment

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…

Continue Reading Vogmask Offers a Stylish Face Mask for Those Living with Immunodeficiencies

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…

Continue Reading X4 Pharmaceuticals Reports Positive Phase 2 Results for WHIM Syndrome Drug
Experimental Treatment for WHIM Syndrome Gets Orphan Drug Designation
jarmoluk / Pixabay

Experimental Treatment for WHIM Syndrome Gets Orphan Drug Designation

According to a story from BioSpace, the biotechnology company X4 Pharmaceuticals recently announced that the US Food and Drug Administration (FDA) has given Orphan Drug designation to the company's investigational…

Continue Reading Experimental Treatment for WHIM Syndrome Gets Orphan Drug Designation
Epigenetic Technology Could Become Useful in Newborn Screening for Primary Immunodeficiency and Monitoring HIV
Rainer_Maiores / Pixabay

Epigenetic Technology Could Become Useful in Newborn Screening for Primary Immunodeficiency and Monitoring HIV

Researchers are exploring a new diagnostic method that could be applied to newborn screening for certain inherited diseases and monitoring of HIV patients. For more detailed information, you can click…

Continue Reading Epigenetic Technology Could Become Useful in Newborn Screening for Primary Immunodeficiency and Monitoring HIV

Interim Results From a Study of a Potential Treatment For WHIM Syndrome Have Been Released

Clinical data from a Phase 2 study of the experimental drug X4P-001-RD for the treatment of WHIM syndrome has been announced at the European Hematology Association’s 23rdCongress in Stockholm. The…

Continue Reading Interim Results From a Study of a Potential Treatment For WHIM Syndrome Have Been Released
A Report on UK Patients with Primary Immunodeficiency Hopes to Inform Medical Professionals About the Conditions
kreatikar / Pixabay

A Report on UK Patients with Primary Immunodeficiency Hopes to Inform Medical Professionals About the Conditions

A primary immunodeficiency report, published in the journal Clinical and Experimental Immunology, examines medical care for primary immunodeficiency patients in the UK. The publication of the report coincides with the…

Continue Reading A Report on UK Patients with Primary Immunodeficiency Hopes to Inform Medical Professionals About the Conditions
A New Study Aims to Improve Diagnoses for People with WHIM Syndrome
Source: Pixabay

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,…

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

Surviving Flu Season with a Rare Disease Featuring Randy Gordon

Rare disease patients remain one of the most underserved medical populations, and they often have to deal with debilitating symptoms or complications that can make daily life difficult. For example,…

Continue Reading Surviving Flu Season with a Rare Disease Featuring Randy Gordon
Screening Test for Navajo Newborns Saves Babies Suffering from Severe Combined Immunodeficiency (SCID)
Source: Pixabay

Screening Test for Navajo Newborns Saves Babies Suffering from Severe Combined Immunodeficiency (SCID)

Joy Littlesunday is just one of many Navajo newborns who were screened and thus saved from severe combined immunodeficiency by a successful bone marrow transplant at UC San Francisco, reported UCSF…

Continue Reading Screening Test for Navajo Newborns Saves Babies Suffering from Severe Combined Immunodeficiency (SCID)
Close Menu