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- PMLiVE

spinal muscular atrophy

- PMLiVE

Novartis’ targeted combination therapy recommended by NICE to treat childhood brain tumours

Gliomas are the most common type of brain cancer in paediatric patients

- PMLiVE

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

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

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Novartis shares positive late-stage results for Fabhalta in rare kidney disease IgA nephropathy

Approximately 25 people per million worldwide are diagnosed with IgAN every year

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Novartis’ blood cancer therapy Kymriah recommended by NICE for routine NHS use

In England, ALL is responsible for around 300 new diagnoses every year in people aged 25 years and under

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Novartis gains rights to Arvinas’ prostate cancer therapy in deal worth over $1bn

The transaction also includes the sale of Arvinas’ preclinical AR-V7 programme

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Novartis shares positive real-world data for Leqvio in atherosclerotic CVD

LDL-C is a causal risk factor for ASCVD and increases the risk of heart disease and stroke

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Novartis’ Fabhalta recommended by CHMP to treat rare blood disorder PNH

Paroxysmal nocturnal haemoglobinuria affects approximately ten to 20 people per million worldwide

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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

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