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Why patient engagement must come first in rare disease trials

Explore why patient engagement is the cornerstone of successful rare disease trials, emphasising the importance of designing studies around the realities of patients' lives to build trust and improve outcomes.

Cuttsy + Cuttsy

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New Cuttsy+Cuttsy research shows why site support matters more than ever

Cuttsy+Cuttsy's upcoming report, 'The site factor: Where protocol meets practice in clinical trials', delves into the critical role of site staff in clinical research, offering insights into their daily challenges,...

Cuttsy + Cuttsy

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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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AstraZeneca and Daiichi Sankyo’s Datroway given FDA Priority Review for breast cancer treatment

Approximately 70% of metastatic triple-negative breast cancer patients are ineligible for immunotherapy, creating an unmet medical need

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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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Genentech reports positive trial results for obesity treatment

The treatment is designed to tackle obesity by reducing appetite and regulating blood sugar

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Gilead’s Trodelvy plus Keytruda shows positive results in metastatic breast cancer

The combination treatment showed a 35% reduction in the risk of disease progression

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Sanofi’s amlitelimab shows positive results in atopic dermatitis

Newly released study data shows consistent reductions in disease severity

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Moderna and Merck announce data from melanoma combination therapy study

Skin cancer is one of the most common cancers in the US, with melanoma accounting for the majority of skin cancer deaths

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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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Vibrant Therapeutics raises $61m in funding to advance intelligent therapeutics pipeline

The financing includes new investors Pfizer Ventures and Apricot Capital, bringing the total capital raised to $100m

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