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

Basliea

- 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

Insights From ISPOR 2025: Breaking the Data Barrier in Rare Diseases – Can AI Bridge the Gap?

Last month, the ISPOR annual meeting in Montreal was brimming with opportunities to explore the latest developments and insights in health economics and outcomes research (HEOR) and market access. In...

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

- PMLiVE

Novartis’ Fabhalta granted FDA approval to treat ultra-rare kidney disease C3G

Approximately half of C3G patients progress to kidney failure within ten years of diagnosis

- PMLiVE

Novartis shares positive phase 3 results for spinal muscular atrophy gene therapy

The company said it is planning to file regulatory applications for OAV101 IT this year

- PMLiVE

NICE recommends Pharming’s Joenja as first treatment for ultra-rare immune disease APDS

Approximately 40 to 50 people in the UK are known to be living with the inherited disorder

- PMLiVE

Amgen’s Uplizna shows promise in phase 3 generalised myasthenia gravis study

Regulatory submissions are expected to be complete in the first half of this year

Subscribe to our email news alerts

Latest content

Latest intelligence

Quick links