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Sanofi’s Xenpozyme not recommended by NICE for ultra-rare disease ASMD

An estimated 40 to 50 people in England are living with the lysosomal storage disorder

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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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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

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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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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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Sanofi’s investigational frexalimab shows promise in relapsing forms of MS

Results from a mid-stage of the candidate were recently published in the NEJM

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Navigating challenges of MedTech research with rare-disease patients

Authored by: Tom Donnelly, MedTech Director and Amanda Pirraglia, Associate Director. Published in Quirk's Magazine.

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