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onasemnogene abeparvovec

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Novartis breaks ground on US radioligand therapy site

The construction is part of the company’s investment in US research and manufacturing

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LEO Pharma to acquire Replay gene therapy platform

The acquisition will advance innovation for patients with rare skin diseases

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MHRA authorises Biogen’s higher-dose Spinraza for SMA treatment

SMA is a rare genetic neuromuscular condition affecting around 1,600 people in the UK

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Shionogi starts phase 2 clinical trial for Pompe Disease

The rare disease affects around one in every 22,000 people worldwide

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PharmaQuest returns: EPA perspectives, through a rare disease lens

By Lottie Sequerra and Emily Morton-West

Amiculum

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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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