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- PMLiVE

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- 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

- PMLiVE

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

The companies will work on therapies for cardiometabolic and rare diseases

Biogen Idec building

Biogen’s Qalsody recommended by CHMP to treat rare form of ALS

Mutations in the SOD1 gene are responsible for approximately 2% of all ALS cases

- PMLiVE

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

- PMLiVE

Enhancing the patient–HCP relationship in rare diseases

By Maggie Lai

AMICULUM

- PMLiVE

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

- PMLiVE

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.

Research Partnership

Biogen Idec building

Biogen receives EC approval for Skyclarys in Friedreich’s ataxia patients

The company gained access to the drug through its $7.3bn acquisition of Reata Pharmaceuticals

- PMLiVE

Sanofi presents positive results for Pompe disease enzyme replacement therapy

The rare genetic disorder is estimated to affect around one in every 40,000 people in the UK

- PMLiVE

Clinigen’s NaviGATE programme reveals knowledge gaps in rare disease community

There are over 7,000 rare diseases that impact more than 300 million people globally

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