How AI is helping solve the labor issue in treating rare diseases

At Web Summit Qatar, AI-powered biotech startups describe how automation, data, and gene editing are filling labor gaps in ...
Fierce Pharma

FDA’s rare pediatric disease voucher program revived by 2026 government funding bill

The Consolidated Appropriations Act of 2026, signed by Trump on Feb. 3, includes a provision reinstating the Mikaela Naylon ...

Officials scramble to contain rare disease after first cases in years: 'The situation is under constant monitoring'

"The situation is under constant monitoring and all necessary public health measures are in place," the Indian Health ...
BioSpace

Rare Pediatric Disease Vouchers Reauthorized, PBM Reform Funded in Narrowly Passed Spending Bill

U.S. President Donald Trump signed a spending package into law Tuesday that reauthorizes the FDA’s previously stalled rare ...
Pharmaceutical Technology on MSN

Rare paediatric disease voucher programme makes long-awaited US return

After more than a year out in the cold, the programme’s reauthorisation is a boost for rare disease therapy developers.
BioSpace

From Awareness to Acceleration: Rare Disease Drug Development Enters a Pivotal Era

Investor enthusiasm and evolving FDA pathways are accelerating rare disease drug development, with ultrarare conditions like ...
Good News Network

Successful World First: Baby Treated with Personalized CRISPR Gene Therapy for Rare Disease is Now ‘Thriving’

Dr. Rebecca Ahrens-Nicklas and Dr. Kiran Musunru crafted a bespoke treatment that has successfully corrected the genetic ...
STAT

As its immunology work draws attention, Sanofi also pushes forward in rare disease

An experimental Sanofi drug helped improve the neurological problems seen with a rare disease, as the company plans to seek ...
STAT

‘Where’s our CRISPR miracle?’

Torie Bosch is the First Opinion editor at STAT. Celena Lozano’s son Benny, who turned 5 in November, loves trains, trucks — anything that goes. He also has a rare disease, PURA syndrome. Earlier this ...

5 Rare Kidney Diseases You May Not Know About

While the leading cause of kidney disease is diabetes, many other factors can lead to kidney disease and failure – including ...
KOAA News5

Falcon family confronts rare disease

FALCON — 10-month-old Emma Jones is battling a rare condition affecting 1 in 18,000 children globally. Her family currently ...
The Print

Number of centres of excellence for rare diseases increased from 8 to 15: Govt

The number of Centres of Excellence under the National Policy for Rare Diseases (NPRD) has been increased from eight to 15 ...