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- PMLiVE

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

- PMLiVE

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

- PMLiVE

Sandoz appoints Armin Metzger as President of new global unit

Metzger was most recently Executive VP, Chief Technical Operations Officer at Ferring Pharmaceuticals

- PMLiVE

US FDA launches ultra-rare diseases framework

The draft guidance specifically discusses genome editing and RNA-based therapies

- PMLiVE

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

- PMLiVE

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

- PMLiVE

Johnson & Johnson’s Darzalex Faspro regimen receives FDA approval for multiple myeloma

Multiple myeloma has an average five-year survival rate of 59.8% and is currently incurable

- PMLiVE

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

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

- PMLiVE

Amgen agrees to lower prescription drug costs in deal with US government

The company has invested $40bn in US R&D and manufacturing in the past seven years

- PMLiVE

Novartis agrees to lower drug prices in deal with US government

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

- PMLiVE

FDA approves first gene therapy for Wiskott-Aldrich syndrome

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

- PMLiVE

FDA launches TEMPO digital health pilot

Digital technologies can improve remote patient care for those with chronic diseases

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