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- PMLiVE

Activartis

- PMLiVE

Sanofi’s Rezurock fast-tracked by NHS for rare chronic graft versus host disease

Around 400 stem cell or bone marrow transplant patients will develop the condition each year in England

- PMLiVE

First UK cancer patients receive Moderna’s experimental mRNA therapy

The Mobilize trial is evaluating mRNA-4359 in cancers including melanoma and lung cancer

- 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

BeiGene’s tislelizumab receives CHMP recommendation for non-small cell lung cancer

Lung cancer is the second most common type of cancer and the leading cause of cancer-related death globally

- PMLiVE

ICR study reveals AstraZeneca’s ceralasertib sensitises tumours to immunotherapy

Results showed that the drug stopped tumour growth in more than half of patients

- 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

Johnson & Johnson’s Tecvayli receives FDA approval for reduced dosing in multiple myeloma patients

More than 35,000 cases of the blood cancer are expected to be diagnosed in the US this year

- PMLiVE

Enhancing the patient–HCP relationship in rare diseases

By Maggie Lai

AMICULUM

- PMLiVE

FDA approves Iovance’s Amtagvi as first cellular therapy for advanced melanoma

Amtagvi is now the first one-time, individualised T cell therapy to be approved in the US for a solid tumour cancer

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