News
How the FDA’s ARC Program Aims to Speed Up Rare Pediatric Disease Treatments
According to a recent FDA listing, ARC is positioned precisely in this space, and the broader regulatory environment is already…
Section
Primary Immunodeficiencies
Curated clinical updates, research breakdowns, and expert perspectives focusing on specific pathways of childhood immune development and pathology.
News
Bridging the Gap: How Proteomics Solves Diagnostic Mysteries in Rare Pediatric Diseases
As CNBC reports, an experimental gene therapy exists that could halt that progression — yet getting it into the patient's hands…
News
Leveraging Targeted Flow Cytometry for Faster Pediatric Immunodeficiency Diagnosis
A 20-year cohort published in Frontiers in Immunology is reinforcing what many of us in pediatric immunology already see at the…
News
Why Routine Childhood Vaccinations Remain Essential for Public Health
Rudaina Banihani, a Newborn & Developmental Pediatrician at Sunnybrook Health Sciences Centre, emphasizes that these diseases…
News
Scaling Precision Oncology: The National Expansion of Australia’s Zero Childhood Cancer Program
As reported by Oncodaily, the Children's Cancer Institute is widening the reach of its flagship Zero Childhood Cancer Program…
News
Advancing Pediatric Hematology and Oncology: Beyond Initial Treatment Decisions
According to Arab Times Online, pediatric hematology and oncology are undergoing rapid developments, with treatment innovation…
News
Strengthening Pediatric Care: Egypt Trains Specialists to Identify Primary Immunodeficiencies
According to the World Health Organization Regional Office for the Eastern Mediterranean, Egypt's Ministry of Health and…
News
Dr. Wen-Yuan Hsieh Named Chief of Pediatric Allergy and Immunology at UT Southwestern
As reported by UT Southwestern Medical Center, physician-scientist Wen-Yuan "Elena" Hsieh, M.D., has been appointed Chief of the…
News
Beyond Survival: Managing Long-Term Immune Health in Pediatric Cancer Survivors
As Childhood Cancer Awareness Month approaches this September, reporting from Newswise puts a striking epidemiological reality in…
News
Adenotonsillectomy Linked to Higher Risk of Pediatric Respiratory Infections
When pediatric teams sit across from families weighing an adenotonsillectomy, the conversation usually centers on obstructive…
News
Single-Cell Mapping Offers New Precision in Predicting Pediatric Crohn's Disease Outcomes
According to Medical Xpress, a new single-cell mapping approach is starting to change that picture, offering an early read on…
News
Mapping T-Cell Gene Circuits to Decode Pediatric Immune Deficiencies
A new Perturb-seq study reported on Medical Xpress gives us, for the first time, a near-complete functional map of the gene…
News
Vitamin D Levels and Immune Resilience in Pediatric Primary Immunodeficiency
According to a recent retrospective cross-sectional study published in MDPI, clinicians reviewing the charts of 132 children…
Primary Immunodeficiencies
First immunology appointment: checklist for parents
When a parent walks into a pediatric immunology clinic for the first time, I can usually tell within the first five minutes…
News
New Clinical Protocol: Verifying SCID Status Before Rotavirus Vaccination
According to updated UK government guidance published on GOV.UK, the live rotavirus vaccine should now only be administered after…
News
Chikungunya in Children: Unique Immune Responses and Clinical Patterns
A new review published in Frontiers consolidates the immunological and clinical evidence on chikungunya fever in children…
News
Newborn Screening for SCID: Closing the Survival Gap in Pediatric Immunology
A new study led by UCSF researchers confirms what many of us in pediatric immunology have hoped to see for years: newborn…
News
Mayo Clinic Joins Federal AEGIS Initiative to Develop Gene-Editing Cures for Rare Immune Disorders
That is why the news from Mayo Clinic — reported via Newswise this week — carries real weight for the families and clinicians…
Primary Immunodeficiencies
WAS treatment: Stem cell transplant vs gene therapy
Allogeneic hematopoietic stem cell transplantation achieves overall survival above 90% in children with Wiskott–Aldrich syndrome…
News
Mapping the Genetic Network Regulating Type I Interferon in Human Monocytes
Published in parallel on PubMed, a second genome-wide study hands pediatric immunologists a sharper atlas for diagnosing the…
News
Navigating Clinical Setbacks in Pediatric Thymus Transplantation
According to CBS Los Angeles, the toddler, Syanne Landron, just turned two in an isolation room on the hospital's fifth floor…
News
Hidden Aggressive Cell Populations Explain Unexpected Relapse in Pediatric Rhabdomyosarcoma
The work, published this week in Cancer Research and reported by Medical Xpress, points to hidden aggressive cell populations…
Primary Immunodeficiencies
ADA-SCID treatment: Enzyme replacement or transplant?
Adenosine deaminase deficiency accounts for approximately 10%–15% of severe combined immunodeficiency cases. The treatment…
Primary Immunodeficiencies
Hyper-IgE syndrome: bypassing eczema diagnostic traps
Hyper-IgE syndrome can begin with a skin problem that looks familiar: severe infantile eczema, a persistent neonatal rash, or…
Primary Immunodeficiencies
Wiskott-Aldrich misdiagnosis: avoiding costly ITP traps
When a young boy presents with bruising, petechiae, nosebleeds, or an unexpectedly low platelet count, immune thrombocytopenia…
Primary Immunodeficiencies
Thymus transplant vs stem cell transplant for athymia
Reported overall survival after hematopoietic stem cell transplantation in congenital athymia is approximately 41%, compared with…
Primary Immunodeficiencies
Congenital neutropenia: G-CSF vs stem cell transplant
Severe congenital neutropenia (SCN) is defined by a persistent absolute neutrophil count (ANC) below 500 cells/µl, usually with…
Primary Immunodeficiencies
MMR vaccine in DiGeorge syndrome: when is it safe?
A child with a 22q11.2 deletion may be listed in the chart as having DiGeorge syndrome, but that label does not answer the MMR…
News
Navigating the 2025 Practice Parameter for Inborn Errors of Immunity
According to a recent National Institutes of Health record, the Practice Parameter for Inborn Errors of Immunity (IEI) was…
News
Genetic Screening for Rare Pediatric Diseases: Separating Clinical Reality from Headlines
According to CNBC, newer genetic-screening methods are being used to identify some rare diseases before symptoms appear, a shift…
Primary Immunodeficiencies
CGD prophylaxis: what to gather before starting therapy
When I sit down with a family after a fresh CGD diagnosis, the most common opening question is not "what is this disease." By the…
News
Why Pediatric Rheumatic Diseases Require a Distinct Genetic Approach
According to a report on research from Children’s Hospital of Philadelphia (CHOP), pediatric-onset rheumatic and other…
Primary Immunodeficiencies
Gene therapy or transplant for Wiskott-Aldrich syndrome
Wiskott–Aldrich syndrome has an estimated incidence of approximately 1 in 250,000 live male births. It is an X-linked inborn…
News
Rare Ventures Secures $25M Grant to Accelerate Pediatric Rare Disease Research
According to TribLIVE, that organization has raised more than $80 million and supported clinical trials, including two genetic…
Primary Immunodeficiencies
Abnormal SCID Screen: A Parent's Diagnostic Roadmap
An abnormal SCID newborn screen is an urgent finding, but it is not the same as a diagnosis of severe combined immunodeficiency…