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There are over 10,000 rare diseases affecting an estimated 300 million people worldwide where 80% are genetic , 95% lack approved treatments and nearly half begin in childhood. ClinicalTrial Manager/Sr. These measures enhance recruitment, retention and overall trial success.
Despite advancements in neuroscience and psychiatry, clinicaltrials for mental health conditions such as major depressive disorder (MDD), schizophrenia, bipolar disorder and generalized anxiety disorder face unique challenges. Finally, psychiatry clinicaltrials must also address the variability in treatment responses.
Written By: Derek Ansel, MS, CCRA, Executive Director, Therapeutic Strategy Lead, Rare Disease Given that 80% of rare diseases have a genetic etiology, genetic implications should be addressed at the onset of a clinical program to support trial enrollment.
Now an international team of researchers has discovered a new genetic variant in people of African ancestries that appears to restrict HIV replication after an infection sets in. Though more research is needed to confirm their findings, the […]
Scientists have found an extremely subtle twist in the genetics of aging cells, one that seems to make them increasingly less functional as time goes on.
Fortunately, advances in clinicalresearch are providing hope for better treatments and outcomes. With the support of global networks like ours at Worldwide ClinicalTrials (Worldwide), autoimmune and bone health research is accelerating, providing new solutions to patients around the world.
Indian healthcare industry is of the view that emergence of genetic diagnostics will be a paradigm shift towards personalized medicine. Through the ever-evolving landscape of healthcare, the role of genetic diagnostics offers personalized insights and proactive solutions for disease management and prevention.
We are witnessing a revolution in healthcare, driven by advances in genetics, Omics, RNA and CRISPR gene-editing technology, to deliver precision and personalised medicine, said Kiran Mazumdar-Shaw, executive chairperson, Biocon and Biocon Biologics. This holds the potential to cure genetic diseases that have plagued families for generations.
Fortunately, advances in clinicalresearch are providing hope for better treatments and outcomes. With the support of global networks like ours at Worldwide ClinicalTrials (Worldwide), autoimmune and bone health research is accelerating, providing new solutions to patients around the world.
The convergence of gene therapies and clinicalresearch is pushing the boundaries of what’s possible in ophthalmic care, offering hope for more effective treatments and potential cures for a range of vision-threatening conditions.
In a recent webinar, Challenges of Drug Development in Early Parkinsons Disease , Tom Babic, MD, PhD , our Vice President of Scientific Solutions in Neuroscience, uses his 40+ years of experience to share invaluable insights into the current state and future directions of early Parkinsons disease (PD) research.
One way to ensure customized disease management is to utilize pharmacogenomics (PGx) in your clinicaltrial. As the study of how an individual’s genes affect their response to drugs, pharmacogenomics is a critical factor in developing effective and safe drugs and dosages in clinicaltrials.
Indian healthcare looks at pharmacogenomics, the study of influence of genetic factors on drug response, in patient care as comprehensive genetic tests gain ground.
When using genetic screening to identify clinicaltrial volunteers, a sponsor’s obligations for further testing and disclosure of results to patients and families are unclear, especially when the results have no impact on medical management. Guidance in this area is much needed. Read the full article! Read the full article!
The leading Australian CRO for biotechs and Frost & Sullivan Asia-Pacific CRO Market Leadership Award winner, Avance Clinical, has expanded its gene technology clinicaltrial services to meet the increasing global demand predicted to reach $17.4 billion by 2023.
Evolving sentiments recognize obesity as a medical condition and lifestyle choice as well as a chronic disease with genetic, psychological, cultural, and environmental causes (Figure 1). These data emphasize the need for precise and meaningful body composition endpoints in T2D and obesity clinicaltrials.
In clinicalresearch, this includes designing trials that elevate the patient voice, reduce barriers to participation, and represent the diverse populations who will ultimately use these medical products. Access to information: Limited resources and educational materials hinder informed decision-making about trial participation.
Pediatric rare disease clinicaltrials are vital to determining the safety and efficacy of medications for children living with a rare disease. About 80 percent of rare diseases have a genetic basis. Most rare diseases are chronic conditions, and many are clinically progressive and potentially fatal. adults).
(Richard Jones/Science Photo Library/Getty Images) Scientists have discovered hundreds of genes that could potentially promote cancer, new research shows. Cancer is typically triggered by some kind of change to our genetic coding that interferes with a cell’s ability to manage its growth.
But before pharmaceutical companies can go to market with a breakthrough drug, they need to ensure safety and efficacy through clinicaltrials. Pharma R&D teams are solving this problem by leveraging the power of artificial intelligence (AI) in clinicaltrials to save time and money.
Making sure clinicaltrials are diverse and representative of the populations they’re studying isn’t just the right thing to do to promote inclusivity and equity; it’s also just good science/clinical scientific practice. Potential barriers that exclude communities from clinicalresearch.
This initiative is particularly impactful for rare diseases and genetic disorders, where traditional endpoints may be infeasible. This shift not only prioritizes patient safety but also ensures that ineffective treatments are identified more quickly.
New research from ETH Zürich in Switzerland could see future wearable devices (with perhaps a few implants and a touch of genetic engineering) boost our health directly. Fitness trackers help you stay healthy by keeping count of your steps and monitoring your heart rate, driving you on to hit those cardio goals.
Scientists from the US, UK, and Uruguay analyzed data from 378,932 people aged 40 to 69 to see how those who are genetically ‘programmed’ to nap regularly fared cognitively […]
Similar genetic changes in two different types of brain cells may contribute to cognitive impairment in schizophrenia and aging. US researchers examined gene expression in more than a million brain cells collected post-mortem from 191 donors.
As DNA holds the genetic code controlling how our bodies grow and are maintained, that’s a serious problem that […] The artificial sweetener sucralose (marketed as Splenda) is widely used and found in products like diet soda and chewing gum.
Diversity, equity and inclusion (DEI) have become important elements in the landscape of clinicalresearch. Xtalks spoke with Stacy Weil, DHI, Senior Vice President, Business Operations and Patient Centricity, Premier Research, to gain insights into incorporating DEI practices and plans in clinicalresearch.
The Foundation for the National Institutes of Health (FNIH) announced this week that the Accelerating Medicines Partnership Bespoke Gene Therapy Consortium (AMP BGTC) has selected eight rare diseases for its clinicaltrial portfolio. Of the more than 10,000 rare diseases, more than 80 percent are caused by genetic defects.
The current research areas of interest in India include precision medicine which treatments based on individual genetic profiles to improve […] The development of innovative therapeutic approaches in healthcare encompass a variety of fields, including gene therapy, personalized medicine, immunotherapy, and advanced biologics.
Around 8% of human DNA is made up of genetic sequences acquired from ancient viruses. Our latest research suggests that some ancient viral DNA sequences in the […]
Generative artificial intelligence (AI) techniques are increasingly being applied in clinicalresearch to enhance various aspects of medical data analysis, diagnosis, treatment planning, and drug discovery. ClinicalTrial Design and Optimization: Generative AI can aid in the design and optimization of clinicaltrials.
One of the more intriguing developments in cancer research in recent years is the growing understanding of clonal hematopoiesis, a phenomenon where blood cells expand from a single clone due to genetic mutations. Clonal hematopoiesis increases in prevalence with age and can precede hematologic malignancies.
The Union ministry of health and family welfare (MoHFW) included primary hyperoxaluria, a rare genetic metabolic disorder that affects kidney and other organ systems, under the National Policy for Rare Diseases (NPRD), 2021, with financial assistance to be provided for transplants.
CSF biomarkers have been a popular area of interest in neurodegenerative disease research with previous studies related to Alzheimer’s and Multiple Sclerosis exploring the potential of their use. In PD clinicalresearch, many biomarkers, such as neurofilament, can be measured within the CSF using a lumbar puncture.
Each year, FDA selects a limited number of clinicaltrials to fund to help sponsors pursue development of medical products for rare diseases and advance their field. In October, FDA announced seven new clinicaltrial grants awarded in fiscal year (FY) 2024 – including one for a Phase 3 trial – totaling $17.2
In the rapidly evolving landscape of clinicalresearch, data has become one of the most valuable assets. ClinicalTrial Management Systems (CTMS) play a pivotal role in managing this data, encompassing everything from patient information and trial protocols to sensitive medical records and regulatory documents.
The 2024 ASCO Annual Meeting from the American Society of Clinical Oncology is a fantastic platform for clinicalresearchers to discuss the latest advancements and challenges in oncology research. Check out this webinar on navigating the landscape of oncology clinicaltrials.
Genetic inheritance is a physical link that tethers our birth parents; one-half of two sets of chromosomes meshing together to form our own unique set of molecular instructions for life.
Drug resistance in DEEs often stems from genetic and molecular factors unique to each syndrome, limiting standard treatments’ effectiveness. Current treatment options remain limited, and many patients have not had opportunities to participate in trials specifically tailored to their condition.
In trials, Casgevy was shown to help prevent episodes of debilitating pain, known as vaso-occlusive crises, that often plague patients affected by sickle cell disease. sickle cell disease community and the millions around the globe who suffer from this genetic illness.
Food and Drug Administration (FDA) has taken steps to address these disparities in clinicaltrial study enrollment over the past two decades, including recent draft guidance issued in June 2024, which mandates diversity action plans for all Phase II and III registrational studies.
As the research and medical community learns more about population variances in disease outcomes, whether as a result of regional genetic profiles, socioeconomic factors, or environmental inputs, it’s become necessary to ensure that clinicalresearchtrials include broad swaths of global participants.
Eli Lilly shared new Phase II trial data showing that its experimental siRNA therapy, lepodisiran, leads to reductions in levels of lipoprotein(a) (Lp(a)) a genetically inherited risk factor for heart disease in adults with elevated levels of the marker. High Lp(a) levels can double or even triple the risk of a heart attack.
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