Gene Therapy for Rare Diseases: Considerations for Both Clinical and Post-Marketing Studies

The AVENIO Tumor Tissue Targeted Kit is a next-generation sequencing (NGS) assay for comprehensive genomic profiling of solid tumors from formalin-fixed paraffin-embedded (FFPE) tissue samples. It contains 17 guideline-aligned biomarkers, including those in the U.S. National Comprehensive Cancer Network (NCCN).

Various neurodegenerative processes result in the development of diseases like Alzheimer’s (AD), Parkinson’s (PD), amyotrophic lateral sclerosis (ALS), and, arguably, multiple sclerosis (MS). Despite years of research, drug discovery initiatives, and promising clinical trials, these diseases remain incurable. But recent studies have suggested common mechanisms underlying these pathologies. Atypical protein assembly resulting in plaque formation is a common pathological finding in both AD and PD, while neuronal death is a primary (ALS) or secondary (MS) hallmark of the disease. For a detailed look at the mechanisms that drive an array of neurodegenerative diseases, The Scientist is bringing together a panel of experts to share their research, discuss current therapeutic approaches, and offer their insights. Come engage with our panel and get the answers you seek.

Due largely to the global COVID-19 pandemic, we are now living in the golden era of mobile health, or “mHealth,” as it has come to be called. The World Health Organization defines mHealth.

Traditional drug manufacturing and drug delivery process development requires a huge time and financial investment. Pharmaceutical and biopharmaceutical leaders are leveraging the power of in silico approaches.