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Why early participant engagement is now a top priority in genetic disease research

pharmaphorum

The DNA of these so-called ‘genetic superheroes’ may contain clues about how to treat severe disease. The idea of ‘genetic superheroes’ and precision medicine is captivating for researchers, but the concept can be abstract and distant for participants. First, start co-development with participants early on in the process.

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Evolution in Hepatitis B and C Virus: Can we expect success similar to Hepatitis C in Hepatitis B as well?

Delveinsight

Due to hepatitis C, most people develop cirrhosis, or scarring of the liver, before liver cancer; about 5–25% of patients with chronic hepatitis C develop cirrhosis over 10–20 years. Research and development in Hepatitis. In contrast, approximately 850,000–2.2 Hurdles in the Hepatitis treatment. Pegylated INF-?2b

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New Study Suggests Pregnant Women Should Avoid Caffeine Altogether. Here’s Why

XTalks

According to a study by researchers at the National Institutes of Health (NIH), moderate caffeine intake could lead to small babies. The research found that even consumption of less than the recommended moderate amount of 200 mg of caffeine per day for adults, which is about two cups of coffee, posed an increased risk to the fetus.

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Adial Pharmaceuticals Provides Update on its ONWARD(TM) Pivotal Phase 3 Trial of AD04 for Treatment of Alcohol Use Disorder

The Pharma Data

(NASDAQ:ADIL; ADILW) , a clinical-stage biopharmaceutical company focused on the development of treatments for addictions, today provided an update on its landmark ONWARD Phase 3 pivotal trial. Adial Pharmaceuticals is a clinical-stage biopharmaceutical company focused on the development of treatments for addictions.

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BridgeBio Pharma’s Nulibry Approved as First Treatment for Molybdenum Cofactor Deficiency Type A

XTalks

BridgeBio is dedicated to developing therapies for genetic diseases with unmet needs. BridgeBio said in an announcement that the novel therapy was developed based on “BridgeBio’s commitment to developing a treatment for MoCD Type A in collaboration with the experts and families in the MoCD Type A community.”

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Biorepositories as a Guiding Resource for Research & Drug Discovery

XTalks

Data generated through high-throughput omics approaches paired with clinical patient records can give rise to remarkably robust datasets to inform and guide disease insights and development of targeted therapeutics. Generation of strong research dataset cohorts must begin with high-quality clinical samples.

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10 Key Learnings from Successful Cellular and Gene Therapy Trials for Rare Diseases

XTalks

“Rare disease clinical trials are complex due to the additional scientific, medical, operational and regulatory requirements of newly emerging advanced therapies, such as gene therapy,” says Dr. Terence Eagleton, MB BS, Senior Medical Director at the global clinical research organization (CRO) Medpace.