Rare Disease Day

News

NICE backs first disease-modifying drug for ARG1 deficiency

Immedica's Loargys is poised to become the first NHS therapy for ultra-rare genetic disorder ARG1 deficiency that can alter the course of the disease.

News

GSK departs Wave's AATD programme

Wave Life Sciences has lost GSK as the partner for its alpha-1 antitrypsin deficiency drug, but other partnered RNA-editing programmes remain active.

News

FDA declines to approve Corcept's rare disorder drug

As the celebrations for the end of 2025 gathered pace, the FDA delivered a year-end blow to Corcept by turning down its new Cushing's drug.

R&D

magnifying glass over a figure in red amid many in black

Novel study designs for rare diseases

Developing treatments for rare diseases poses many unique challenges that cannot be addressed by traditional study designs.

Digital

Eric Luellen and Jonah Comstock interview at AAPS

AI-powered drug discovery is outpacing regulators, with Eric...

During the AAPS National Biotech Conference 2025, pharmaphorum’s Jonah Comstock sat down with Eric Luellen, the founder and CTIO of Turing Biosciences

R&D