Pharmafile Logo
  • - PMLiVE
  • Diversity, Equity and Inclusion
  • - PMLiVE
  • Communiqué 53 Cover Image

polycythaemia vera

- PMLiVE

UCB’s Kygevvi receives positive CHMP opinion for thymidine kinase 2 deficiency

TK2d is an extremely rare disease characterised by severe and progressive muscle weakness

- PMLiVE

Sanofi announces positive results for venglustat in type 3 Gaucher disease

There are currently no approved treatments for the neurological symptoms of this disease

- PMLiVE

Takeda’s zasocitinib demonstrates positive results in plaque psoriasis

Around 64 million people worldwide live with psoriasis

- PMLiVE

Havas Life London unveils AI awareness campaign

The campaign highlights the dangers of AI misinformation in rare disease

- PMLiVE

FDA approves first gene therapy for Wiskott-Aldrich syndrome

Symptoms of the rare disease include bleeding, eczema and frequent infections

- PMLiVE

AstraZeneca announces $2bn investment in US manufacturing

The new and expanded facilities will support production of rare disease drug treatments

- PMLiVE

AstraZeneca reports positive results from rare disease trial

HES is a group of rare disorders characterised by persistently elevated levels of white blood cells

- PMLiVE

BMS, Takeda and Astex join AI training consortium

The initiative focuses on training AI models to aid in drug discovery and development

EU flag

CHMP recommends tablet formulation of BeOne’s targeted cancer drug Brukinsa

The new formulation would allow blood cancer patients to halve their daily pill intake

- PMLiVE

Takeda receives EC approval for Adcetris combination in Hodgkin lymphoma

The decision marks the second approval for an Adcetris-based regimen for front-line Hodgkin lymphoma

- PMLiVE

Takeda’s Adzynma approved by MHRA for rare blood clotting disorder cTTP

The enzyme replacement therapy is now the first treatment approved in the UK for this patient population

- PMLiVE

J&J/Protagonist’s icotrokinra shows promise in difficult-to-treat plaque psoriasis

An estimated eight million people in the US are living with the immune-mediated skin disease

Subscribe to our email news alerts

Latest content

Latest intelligence

Quick links