Pharmafile Logo
T40 2022 Cover
Diversity, Equity and Inclusion
Communiqué 53 Cover Image
- PMLiVE

spinal muscular atrophy

EU flag

EMA recommends revoking marketing authorisation for Novartis’ sickle cell disease drug

The EU regulator concluded that the benefits of Adakveo did not outweigh its risks

- PMLiVE

FDA approves Blueprint Medicines’ Ayvakit for indolent systemic mastocytosis

Ayvakit is now the first and only approved medicine designed to treat the underlying cause of the disease

- PMLiVE

Novartis’ Cosentyx recommended by CHMP for hidradenitis suppurativa

An estimated one in 100 people globally are affected by the inflammatory skin disease

- PMLiVE

Novartis announces positive long-term data for relapsing MS treatment Kesimpta

Approximately 85% of MS patients are initially diagnosed with relapsing forms of the disease

- PMLiVE

NHS England announces rare genetic disorder fast-tracking service

Patients with inherited white matter disorders will benefit from earlier diagnosis and specialist care

Rare Diseases on Medscape Education: Pustular Psoriasis

Rare Disease Day may be over but our committment to advancing outcomes for patients continues. Watch this video to understand a patient's journey with pustular psoriasis.To learn more about Medscape's rare...

Medscape Education Global

Rare Diseases on Medscape Education: Long-Chain Fatty Acid Oxidation Disorder

Rare Disease Day may be over but our committment to advancing outcomes for patients continues. Listen to a mother explain her daughter's journey with a long-chain fatty acid oxidation disorder.To...

Medscape Education Global

- PMLiVE

AstraZeneca announces major expansion of Canadian research footprint

The investment includes the creation of 500 jobs and a new rare disease research hub

- PMLiVE

Rare Disease Day 2023 – raising awareness around the world

Around one in 17 people will be affected by a rare disease at some point in their lives

- PMLiVE

UK government announces new rare diseases action plan for England

The framework lists 13 actions that have been developed with the rare disease community

Medscape Education: Rare Diseases PAH

Today is Rare Disease Day, February 28.Hear from Helen as she describes in her own words what it’s like to live with PAH, and her perspective as a patient.  To learn...

Medscape Education Global

Medscape Education and M4RD: Together Caring for Rare Disease

We are excited to finally share our news-style video ' ', developed in partnership with Medics 4 Rare Diseases (M4RD) and produced by Genetic Alliance UK and ITN Business.For more information on...

Medscape Education Global

Subscribe to our email news alerts

Latest jobs from #PharmaRole

Latest content

Latest intelligence

Quick links