As Rare Disease Day is commemorated on 28 February, experts discuss the barriers to gene therapy access for some rare ...

Teesside University's National Horizons Centre (NHC) is committed to turning groundbreaking ideas into practical solutions.

The CHMP has recommended the approval of AbbVie's upadacitinib (Rinvoq) for treating adults with giant cell arteritis (GCA).

With issues in therapy delivery and a barren trial landscape, short-term chances for a commercial galactosemia treatment are ...

A survey has shown that pharma companies have low confidence their rare disease medicines can launch in the UK.

Medigene and EpimAb Biotherapeutics have signed a co-development agreement involving T cell receptor (TCR)-guided T cell ...

The FDA has accepted Sobi’s sBLA for Gamifant for haemophagocytic lymphohistiocytosis (HLH)/MAS associated with Still’s ...

Genitourinary Cancer Symposium 2025, held on 13-15 February, final results from the randomised, multicentre, double-blind, ...

Alltrna’s CEO Michelle Warner said that the approach allows for basket trials that can group patients with different diseases ...

Zevra has sold its US Food and Drug Administration (FDA) priority review voucher (PRV) for $150m to an undisclosed party.

New EU Clinical Trial Regulations simplify international trials while offering the means to counter drug shortages for trials ...

Deal numbers dropped in January this year compared to last, with outbound deals trumping inbound transactions.