Remove tag patient-flow
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Grand Rounds November 18, 2022: The FIRST-ABC Pragmatic Trials of Non-Invasive Respiratory Support In Children (Padmanabhan Ramnarayan, MBBS, MD, FRCPCH, FFICM)

Rethinking Clinical Trials

Sick children, when they are acutely ill, start with standard oxygen therapy, which escalates to noninvasive respiratory support (CPAP/BIPAP or high flow nasal cannula) before invasive ventilation support in the ICU. acutely ill patients) and step-down, which is the extubation or post-extubation part of the care pathway.

Trials 130
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Orphan drugs and where to launch them: The keys to Europe’s forgotten territories

Pharmaceutical Technology

Historically, the pharmaceutical industry has failed to meet the needs of this patient population. Most famously, the US passed its Orphan Drug Act in 1983, providing innovators with financial motivation to develop orphan drugs and meet the needs of these forgotten patients. Efforts have been made to change this.

Drugs 147
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Grand Rounds February 17, 2023: The Heartline Trial: A New Paradigm in Conducting Virtual Clinical Trials (C. Michael Gibson, MS, MD)

Rethinking Clinical Trials

The Heartline Trial has moved to a new form of information sharing, one that is open source, innovates from without, knowledge flows to and from all via the internet, people can share information, retaining credit for it but sharing it freely. The biggest change is that the patient now has a seat at the table.

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Perfusion MRI and Other Perfusion Imaging Techniques for Clinical Trials

XTalks

Currently, MRI-based perfusion imaging is also being applied to measure changes in inflammation in patients with rheumatoid arthritis and osteoarthritis. Representative images collected by DCE-MRI of a patient with adenoid cystic carcinoma that show a decrease in tumor perfusion after exposure to AG-013736, an oral angiogenesis inhibitor.

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Epigenetics discovery could lead to new class of cancer drugs

Drug Discovery World

The study shows that H3K4me3 ensures genes are transcribed and activated at the right time in a controlled manner, like a set of traffic lights regulating the flow of cars on a busy road. But, until now, scientists lacked an understanding of what the chemical tag does, despite many years of research.

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Lyfgenia and Casgevy Become First FDA-Approved Gene Therapies for Sickle Cell Disease

XTalks

The sickled red blood cells obstruct flow in blood vessels, limiting oxygen delivery to tissues in the body, which leads to severe pain and organ damage called vaso-occlusive events (VOEs) or vaso-occlusive crises (VOCs). It can also make patients more susceptible to complications like anemia, jaundice, infections and gallstones.

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No sign of stopping: The rise of cell and gene therapy

Drug Discovery World

Large pharma involvement in advanced therapies has seen ebbs and flows, with AstraZeneca taking a larger stake in the market and Takeda developing its automation and digital capabilities, but also with GSK letting go of its CGT projects, along with most of the staff involved in its R&D. But are these price tags a barrier to access?