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complement 3 glomerulopathy

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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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FDA gives Rare Paediatric Disease designation to Satellite Bio for urea cycle disorders

The mortality rates of UCDs exceeds 25%, with no curative treatment available for newborns

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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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BMS’ Opdivo gets expanded FDA and EU approval for Hodgkin lymphoma

This type of cancer is the most common one diagnosed in teenagers

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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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US FDA launches ultra-rare diseases framework

The draft guidance specifically discusses genome editing and RNA-based therapies

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PureTech’s deupirfenidone gets FDA and EC Orphan Drug Designations for idiopathic pulmonary fibrosis

Rare diseases are defined as conditions affecting fewer than 200,000 people in the US or fewer than five in 10,000 individuals in the European Union

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Diabeloop’s DBLG2 receives FDA and CE clearance for automated insulin delivery without meal input

While a lack of meal announcements can lead to lower glycaemic control, it reduces the mental burden for those with type 1 diabetes

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