Please provide your email address to receive an email when new articles are posted on . There is no current treatment for children with APDS aged 4 to 11 years. Diagnostic delays have a mean of 7 ...

A Prescription Drug User Fee Act target date of January 31, 2026 has been set for the application. The Food and Drug Administration (FDA) has accepted for Priority Review the supplemental New Drug ...

If approved, leniolisib will be first and only treatment indicated for children with activated phosphoinositide 3-kinase delta (PI3Kδ) syndrome (APDS), a rareprimary immunodeficiency Decision based on ...

In the realm of advanced electronics and photonics, compound semiconductors are the silent enablers of breakthrough performance in many applications. Built from materials like indium gallium arsenide ...

A novel method has been developed to help rapidly diagnose rare genetic diseases. A new laboratory method developed by researchers at Columbia University Vagelos College of Physicians and Surgeons (NY ...

A teenager who lost four family members to a rare genetic condition has become the first person in Europe to receive a “life-changing” drug after it was approved for use on the NHS. Mary Catchpole, 19 ...

A teenager who lost her mother, aunt and uncle to a genetic condition has become the first person in Europe to receive a “life-changing” drug after it was approved for use on the NHS. Mary Catchpole, ...

A teenager who lost her mother, aunt and uncle to a genetic condition is the first person in Europe to receive a "life-changing" drug. Mary Catchpole, 19, from Great Yarmouth, suffers from a rare ...

A teenager from Norfolk has become the first patient in Europe to be given a newly licensed treatment which could potentially cure her life-threatening, inherited disorder. Mary Catchpole, 19, lost ...

Mary Catchpole is the first person to benefit from a new treatment for a rare condition affecting her family A teenager from Norfolk has become the first patient in Europe to be given a newly licensed ...