clinical trial Archives

Rare disease drug development: Time for a new approach?

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A rare disease is one that affects a small percentage of the population.

A limited patient population is a key challenge in rare disease drug development. Professor Shein-Chung Chow of Duke University School of Medicine, USA and his research partners explore innovative approaches to overcome this challenge. These include using external control, selecting appropriate study endpoints, and justifying sample size based on probability statements. They propose the use of a complex innovative two-stage […]

Conquering sunlight sensitivity: Dersimelagon’s promise in photodermatoses

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Dersimelagon in Erythropoietic Protoporphyrias

Two rare genetic dermatological conditions, erythropoietic protoporphyria and X-linked protoporphyria, bring about excruciating pain and skin damage upon exposure to sunlight. In groundbreaking research, a new medication called dersimelagon is addressing these conditions. Led by Dr Kirstine Belongie and her expert team at Mitsubishi Tanabe Pharma America, a clinical trial was conducted to evaluate dersimelagon’s impact on the onset time […]

Clostridium difficile infection: Risk factors and potential vaccines

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Antibiotics treatment is one of the main factors of risk for developing Clostridium difficile (C. diff) infection, a condition responsible for nearly 500,000 infections and 30,000 deaths each year in the U.S. alone. Dr Joseph Shiloach, Dr Ashish Sharma and their collaborators at the Biotechnology Core Laboratory of the National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK) optimised […]

Fragment-based design of isoquinoline derivatives as anti-inflammatory drugs

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Dr Masakazu Atobe and his colleagues have developed a methodology that can be applied to the discovery of effective drugs.

Fragment-based drug discovery (FBDD) is a well-established high-throughput screening method for drug generation. Fragment molecules used in FBDD tend to be structurally less complex than the compounds used in conventional high-throughput screening techniques. Dr Masakazu Atobe from Asahi Kasei Pharma Corporation in Japan and his colleagues have developed a methodology that can be applied to the discovery of effective drugs […]

A new weapon to boost cancer immunotherapy

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The 4SC team monitors the expression of relevant genes in the tumours of patients before and after administration of domatinostat.

In recent years, immunooncology has paved new avenues for effective treatment across cancer types. In addition to cytotoxic chemotherapy, we can now harness the power of the human immune response against malignant cells. However, immunotherapy is not perfect, and as always, cancer has mechanisms to evade these attacks. Dr Svetlana Hamm, Head of Research and Translational Medicine at 4SC in […]

Treating low testosterone in males: A nasal gel shows promising results

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Natesto increases testosterone into normal range.

Ranjith Ramasamy is an Associate Professor in the Department of Urology, University of Miami. His clinical work focuses on treating male infertility and sexual dysfunction, and he is involved in several research projects. One such project investigated a novel treatment for low testosterone, a nasal gel known as Natesto. An initial clinical trial showed that Natesto is a promising treatment […]

Strengthening the case for goal directed fluid therapy

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Intravenous (IV) fluids are critical for maintaining hydration and blood circulation during major abdominal surgery. The right amount of fluids can help prevent complications and speed up postoperative recovery, but finding that sweet spot can be challenging. To help narrow the field, Beijing Tongren Hospital Department of Anesthesiology Vice-Chief Anesthesiologist, Dr Yanxia Sun and her team are running a clinical […]