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Zolgensma

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Novartis to acquire Excellergy in deal worth up to $2bn

Exl-111, a potential treatment included in the deal, is a central factor in many allergic diseases

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Scotland becomes first part of UK to screen all newborn babies for SMA

The screening is part of a two-year evaluation funded by the Scottish government and Novartis

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Novartis agrees on potential $3bn deal to buy Synnovations’s breast cancer treatment

The oral drug is currently being evaluated for breast cancer and other advanced solid tumours in a phase 1/2 study

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UCB’s Kygevvi receives positive CHMP opinion for thymidine kinase 2 deficiency

TK2d is an extremely rare disease characterised by severe and progressive muscle weakness

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Sanofi announces positive results for venglustat in type 3 Gaucher disease

There are currently no approved treatments for the neurological symptoms of this disease

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Novartis’ ianalumab receives FDA Breakthrough Therapy designation for Sjögren’s disease

Approximately half of those with Sjögren’s disease are thought to be undiagnosed

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Novartis agrees to lower drug prices in deal with US government

In 2025, the pharma company announced a $23bn US investment commitment

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Havas Life London unveils AI awareness campaign

The campaign highlights the dangers of AI misinformation in rare disease

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FDA approves first gene therapy for Wiskott-Aldrich syndrome

Symptoms of the rare disease include bleeding, eczema and frequent infections

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Novartis’ Itvisma gets FDA approval for spinal muscular atrophy treatment

Around 9,000 people in the US currently live with SMA

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Novartis announces plans for new US manufacturing hub

The US hub is expected to create 700 new jobs by 2030

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AstraZeneca announces $2bn investment in US manufacturing

The new and expanded facilities will support production of rare disease drug treatments

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