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Sarah Avent

- PMLiVE

Sanofi presents positive results for rilzabrutinib in chronic spontaneous urticaria

Phase 3 development of the treatment is expected to commence later this year

- 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

Shaping up for launch in ’24: three areas to explore for pharma

By Oli Hudson

- 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

CHMP recommends Travere and CSL Vifor’s Filspari to treat rare kidney disease IgAN

The rare form of kidney disease is estimated to affect around 25 per million adults worldwide

- PMLiVE

The pharma trailblazer’s skill set: forged in rare diseases

There is one very special, overlooked area, in large organisations and small, that builds skills like no other: rare diseases

- PMLiVE

Rare diseases – driving further improvements in patient access

Despite significant strides, many rare disease patients have no licensed treatment options and face high levels of unmet need

- PMLiVE

Overcoming challenges in rare disease research with patients

By Beverly Romero

- PMLiVE

FDA approves Allecra’s combination antibiotic Exblifep for complicated UTIs

Complicated UTIs account for approximately 19% of all healthcare-associated infections

- 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

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