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

EURORDIS

- PMLiVE

Amgen’s Uplizna shows promise in rare fibroinflammatory disease IgG4-RD

The company is planning to file for US approval of the CD19-targeted drug in this patient population

Biogen Idec building

Biogen to expand immunology and rare disease portfolio with $1.8bn HI-Bio acquisition

The company will gain access to HI-Bio’s lead asset for immune-mediated and rare diseases

- PMLiVE

AstraZeneca unveils plans to deliver $80bn in total revenue by 2030

The company expects to launch 20 new medicines by the end of the decade

EU flag

European Parliament approves pharma reform compromise for equal access to new medicines

The new proposal will be finalised after the EU elections take place in June 2024

- PMLiVE

AstraZeneca expands rare disease pipeline with $1.05bn Amolyt Pharma acquisition

The deal gives the company access to a late-stage candidate being evaluated for hypoparathyroidism

- PMLiVE

Novartis shares positive results for spinal muscular atrophy gene therapy Zolgensma

The company said the results support the use of the one-time therapy in older and heavier children

- PMLiVE

Radboud researchers use new method to improve diagnosis of rare diseases

Approximately 7,000 rare diseases collectively impact up to 5.9% of the global population

- PMLiVE

OneChain-led consortium granted €1.9m to advance CAR-T therapy for rare leukaemia

Acute lymphoblastic leukaemia has an incidence of around 1.5 cases per 100,000 people

- PMLiVE

Clinigen launches ‘What is Possible?’ campaign on Rare Disease Day to accelerate access to medicines

There are over 7,000 rare diseases impacting more than 300 million people globally

- PMLiVE

Novo Nordisk and Neomorph enter molecular glue degrader partnership worth over $1.4bn

The companies will work on therapies for cardiometabolic and rare diseases

- PMLiVE

Alexion launches ‘colourUp4RARE challenge’ to mark Rare Disease Day 2024

The campaign is aimed at raising awareness of how to improve quality of life for patients

- PMLiVE

Enhancing the patient–HCP relationship in rare diseases

By Maggie Lai

AMICULUM

Subscribe to our email news alerts

Latest jobs from #PharmaRole

Latest content

Latest intelligence

Quick links