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

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

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

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

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

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

The MSSG solidarity mechanism enables medicine sharing between EU states, with companies now required to report on drug ...

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

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

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

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

The FDA has granted fast track designation to Pyxis oncology’s PYX-201 for the treatment of adults with R/M HNSCC.