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onasemnogene abeparvovec

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Novartis announces trial results for remibrutinib in relapsing multiple sclerosis

MS affects nearly three million people worldwide and has three main types

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LEO Pharma acquires rights for dersimelagon from Tanabe Pharma

The company will pay $435m in up front and near-term milestone payments

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Novartis’ Pluvicto approved by FDA for metastatic hormone-sensitive prostate cancer

More than 186,000 men are diagnosed each year with mHSPC globally

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Novartis to acquire Myricx Bio in deal worth $1.5bn

Myricx, a UK-based biotech, is developing a new class of antibody-drug conjugates

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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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