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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. Reference: Chung DC, et al.

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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. In our experience of running 20+ precision medicine studies, there are a few design principles that every research programme – from biobanks to clinical trials – should consider. Giving participants something in return.

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Type 1 Diabetes Vaccine: Does Diamyd Have a Winning Formula?

XTalks

A vaccine developed by Swedish company Diamyd Medical has demonstrated significant treatment efficacy in a predefined genetic subgroup of individuals with type 1 diabetes in a Phase IIb clinical trial. In the trial, the vaccine was injected directly into the lymph nodes of type 1 diabetic individuals. The DIAGNODE-2 Trial.

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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. 40% of expected Trial patient screenings have been completed and the Trial is more than 25% enrolled relative to planned enrollment.

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Top 30 New Medical Devices of 2024

XTalks

The liquid portion of the blood (serum or plasma) containing antibodies is separated and tested using the Liason Biotrin parvovirus B19 IgG plus test on a Liason XL analyzer. In a clinical trial, participants demonstrated a 98% success rate after six months post implantation, with arteries widened successfully and no stent fractures observed.

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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

Even though clinical trial results were promising, adverse events and serious complications were observed. Hepatitis C treatment options, without a doubt, have improved significantly, including pan-genotypic medications that are used to treat all genotypes and subtypes. Both drugs resulted in inhibition of HCV replication.

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HFpEF vs. HFrEF: How To Improve Heart Failure Drug Development

XTalks

Late-stage clinical trial failures are largely to blame for the lack of effective treatment options for patients with HFpEF, and poor five-year survival rates make this an area of great unmet need. A lack of efficacy is the greatest reason for clinical trial failures, especially in Phase II and Phase III,” says Dr. Tyl.