Healio

FDA accepts supplemental new drug application for leniolisib for children with APDS

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 ...
Monthly Prescribing Reference

Leniolisib Gets Priority Review for Young Children With APDS

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 ...
PharmiWeb

Pharming Group announces U.S. FDA acceptance and Priority Review of supplemental New Drug Application for leniolisib in children with APDS aged 4 to 11 years

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 ...
GitHub

SparkFun APDS9960 RGB and Gesture Sensor Arduino Library

* **/examples** - Example sketches for the library (.ino). Run these from the Arduino IDE. * **/src** - Source files for the library (.cpp, .h). * **library ...
BioTechniques

The method revolutionizing the diagnosis of rare genetic diseases

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 ...
The Independent

Breakthrough for teen who lost four family members to inherited condition

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 ...
Sky

'Life-changing' drug for teenager who lost mother to genetic condition

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 ...
BBC

Drug for rare disease brings 'joy and hope'

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 ...
BBC

Drug for rare disease brings 'joy and hope'

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 ...
pharmaphorum

First drug for rare disease APDS cleared for NHS use

Pharming's Joenja has become the first disease-modifying drug that can be used by the NHS to treat activated phosphoinositide 3-kinase delta (PI3Kδ) syndrome (APDS), an ultra-rare immunodeficiency ...
CNX Software

ANAVI Miracle Emitter – A WiFi and BLE RGB LED controller compatible with Home Assistant, WLED firmware (Crowdfunding)

ANAVI Miracle Emitter is an open-source hardware ESP32-C3 WiFi and BLE controller designed to control 5V addressable RGB LED strips, which works with Home Assistant over MQTT and also supports the ...
PMLiVE

NICE recommends Pharming’s Joenja as first treatment for ultra-rare immune disease APDS

The National Institute for Health and Care Excellence (NICE) has recommended Pharming’s Joenja (leniolisib) to treat patients with the ultra-rare immune disease, activated phosphoinositide 3-kinase ...