Pediatric gene therapy trials: the enrollment roadmap
For families considering a pediatric gene therapy clinical trial, the most difficult part is often not finding a study.
For families considering a pediatric gene therapy clinical trial, the most difficult part is often not finding a study.
The 2025 STOP-JIA trial results confirmed what many of us in pediatric rheumatology had suspected for years: earlier…
A pneumococcal vaccine schedule for an immunocompromised child is defined by two variables: the child’s underlying risk…
The conversation I keep having with genetics counselors goes something like this: a couple arrives after losing a first…
Intravenous and subcutaneous immunoglobulin replacement constitute the two established administration routes for…
Approximately 50 to 60 percent of positive results on standard skin prick tests (SPT) and serum-specific IgE (sIgE)…
A single dose of a long-acting monoclonal antibody such as nirsevimab provides passive protection against severe…
When a child has recurrent, unusually severe, or difficult-to-explain infections, the clinical challenge is not simply…
The deferral question lands on my desk roughly once a week. A family has a child on monthly IVIG for a primary antibody…
For moderate-to-severe pediatric atopic dermatitis, the treatment distinction is no longer simply topical therapy…
When parents or clinicians consider separating combination vaccines into individual injections, the discussion often…
The 2017 FDA approval of tisagenlecleucel for pediatric and young adult relapsed/refractory B-cell ALL was supposed to…
For a child, stepping down asthma controller medication is usually considered only after at least six months of stable…
Wiskott–Aldrich syndrome has an estimated incidence of approximately 1 in 250,000 live male births. It is an X-linked…
Vaccine titer testing for immunocompromised children can answer an important clinical question: has the child produced…
In my experience running pediatric immunology cohorts, the difference between a clean flow cytometry readout and a…
The 2026 American College of Rheumatology guidelines materially change several juvenile idiopathic arthritis treatment…
Genetic testing can clarify a child’s immune disorder, change the management pathway, and sometimes prevent years of…
In July 2026, the BMJ published the SPIRIT-C 2026 and CONSORT-C 2026 extensions — a set of new reporting expectations…
An abnormal SCID newborn screen is an urgent finding, but it is not the same as a diagnosis of severe combined…
For children with persistent allergic rhinitis, antihistamines and nasal sprays can control symptoms, but they do not…
A 76.9% reduction in severe acute respiratory infection (SARI) among infants was reported in one study when household…