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First patient dosed in ulcerative colitis drug trial

Drug Discovery World

Direct Biologics uses extracellular vesicles (EVs) derived from bone marrow mesenchymal stem cells (MSCs) to address multiple disease indications. The post First patient dosed in ulcerative colitis drug trial appeared first on Drug Discovery World (DDW).

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Six advances in drug discovery for MND / ALS

Drug Discovery World

Ahead of Motor Neurone Disease (MND) Awareness Day on 21 June, we take a look back over the latest developments in the treatment of MND and amyotrophic lateral sclerosis (ALS). A new UK research partnership In June, UK charity LifeArc pledged £1.5

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Personalising whole genome sequencing doubles diagnosis of rare diseases 

Drug Discovery World

A new study led by Medical Research Council-funded researchers from UCL has found that tailoring the analysis of whole genome sequencing to individual patients could double the diagnostic rates of rare diseases. . It also detected potential disease-causing variants in a further 3.9% Context . The study . of patients. .

Disease 130
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ASH23: CAR-T for autoimmune disease, real-world questions and sickle cell’s social media mentions

BioPharma Drive: Drug Pricing

Study results disclosed at the American Society of Hematology meeting Saturday suggested potential for cell therapy in lupus and pointed out drug trial limitations.

Disease 124
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Mathematical model can speed up Alzheimer’s drug development

Drug Discovery World

Researchers have created a mathematical model that can accurately depict Alzheimer’s disease progression in individual patients, paving the way for personalised treatment and faster development of therapies.

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This week in drug discovery (30 Oct-3 Nov)  

Drug Discovery World

News round-up for 30 October – 3 November by DDW Editor Reece Armstrong My news highlights this week feature a variety of exciting clinical trial results and launches that are examining therapies for much-needed disease areas, new drug approvals and even a space mission with a pharma twist.

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An Evolving Regulatory Environment for Rare and Orphan Diseases

Advarra

A rare, or orphan, disease by definition affects a small percentage of the population — fewer than 200,000 people in the U.S. But the numbers add up, and taken together, rare diseases impact an estimated 30 million Americans. Before 1983, only 38 orphan drugs had received U.S. Food and Drug Administration (FDA) approval.

Disease 52