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

Genomics England

- 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

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

NHS programme to give head and neck cancer patients fast-track access to mRNA vaccine

More than 11,000 people are diagnosed with head and neck cancers every year in England

Navigating NHS Market Access: Policy Change and Opportunity in England With Sarah Everest Ford

Navigating NHS Market Access: Policy Change and Opportunity in England

With the changes at NHS England and the publishing of the 10-Year Plan, there is a lot of talk of change in the NHS in England. But what do these...

Petauri Evidence

- PMLiVE

Digital Transformation in the NHS’s 10-Year Plan: Techno-Optimism and the Limits of Efficiency

Here, Evelyne Priestman (Senior Consultant – HEOR) and Mary Baker (Senior Medical Writer) at Petauri Evidence explore the National Health Service (NHS) in England’s newly unveiled 10-year plan through the...

Petauri Evidence

- PMLiVE

Live Q&A: Beyond NHS England – Navigating changes at NHSE and DHSC

What impact can we expect from the abolition of NHS England (NHSE) and changes at the Department of Health and Social Care (DHSC)? What do these changes mean for your...

Petauri Evidence

- PMLiVE

Amgen’s Uplizna approved by FDA as first treatment for rare disease IgG4-RD

Immunoglobulin G4-related disease affects approximately 20,000 people in the US

- PMLiVE

FDA approves Soleno’s Vykat XR as first treatment for hyperphagia in Prader-Willi syndrome

The rare genetic disorder affects an estimated one in every 15,000 newborns in the US

Subscribe to our email news alerts

Latest content

Latest intelligence

Quick links