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Isis Pharmacueticals

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Roche’s ALK inhibitor Alecensa receives FDA approval to treat early-stage NSCLC

Approximately half of all patients with early lung cancer experience cancer recurrence following surgery

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Roche reveals promising phase 3 results for subcutaneous Ocrevus in progressive and relapsing MS

The chronic neurological condition affects more than 2.8 million people worldwide

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Roche receives FDA breakthrough device designation for Alzheimer’s disease blood test

Around 55 million people globally are living with dementia, which is estimated to rise to 139 million by 2050

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Roche’s Columvi combination shows promise as second-line DLBCL therapy

Approximately 160,000 people worldwide are diagnosed with DLBC every year

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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

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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

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

The companies will work on therapies for cardiometabolic and rare diseases

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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

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Enhancing the patient–HCP relationship in rare diseases

By Maggie Lai

AMICULUM

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