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gantenerumab

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The draft guidance specifically discusses genome editing and RNA-based therapies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

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Eisai and Biogen’s subcutaneous Leqembi given FDA Priority Review for early Alzheimer’s

Administering the drug subcutaneously rather than intravenously showed similar clinical benefits for patients

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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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FDA approves first gene therapy for Wiskott-Aldrich syndrome

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

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Lilly and Indiana University collaborate to expand clinical trial access

The five-year agreement includes a focus on R&D for Alzheimer’s disease

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FDA launches TEMPO digital health pilot

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

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Roche’s Columvi improves survival in large B-cell lymphoma

Approximately 160,000 people are diagnosed with this aggressive form of cancer each year

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Novo Nordisk presents new findings on semaglutide in Alzheimer’s disease

While the trials did not meet their primary endpoints, they highlighted important biomarker findings

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Vandria announces positive phase 1 data for Alzheimer’s treatment

Trial data showed that VNA-318 has the ability to penetrate the brain

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