Rare Disease

Patients

ASCO 2026: New hope in AL amyloidosis, with Dr Hans Lee

A phase 1/2 trial in AL amyloidosis saw 100% of patients obtaining at least a very good partial response with 90% obtaining a complete response.

Market Access

Young girl gives high five to her female doctor, mum also in the consult room

A step forward, but not the finish line: What NICE’s QALY up...

Healthcare in the UK is at an inflection point.

R&D

Rare disease at an inflection point: Why the next wave will ...

As rare disease becomes more competitive, information parity is disappearing.

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

Why it is more important than ever to optimise and innovate ...

Rare disease research is vital to improve the lives of hundreds of millions of people worldwide.

R&D

On incentivisation and investment in rare disease R&D

To mark Rare Disease Day, pharmaphorum spoke with Paola Pozzi, partner of telethon strategy at Sofinnova Partners, about the outlook for investment in this field of research.