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UKRI appoints iiCON to deliver £1.5m initiative to tackle infection transmission

The project will aim to accelerate solutions that leverage technologies such as AI and novel diagnostics

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FDA awards Orphan Drug Designation to SynaptixBio for rare brain disease therapy

Isolated hypomyelination is a form of TUBB4A leukodystrophy that has been identified relatively recently

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Sanofi presents positive results for rilzabrutinib in chronic spontaneous urticaria

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

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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

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

By Oli Hudson

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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

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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

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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

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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

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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

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Overcoming challenges in rare disease research with patients

By Beverly Romero

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FDA approves Allecra’s combination antibiotic Exblifep for complicated UTIs

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

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