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Most treatments being pursued today to protect against Alzheimer's disease focus on amyloid plaques and tau tangles that accumulate in the brain, but new research points to a novel -- and noble -- approach: using Xenon gas. A phase 1 clinical trial of the treatment in healthy volunteers will begin in early 2025.
The need for more research into rare diseases is clear, but researchers face significant challenges. Fortunately, technology has created opportunities to innovate in clinical trials for rare diseases.
Tests, in mice, of a drug developed by the researchers showed that regulatory T cells can be attracted to specific body parts, boosted in number, and activated to suppress immune response and rebuild tissue. Clinical trials in humans are now planned.
Researchers have used the genome-editing tool CRISPR-Cas to create disease resistant rice plants, according to a new study. Small-scale field trials in China show that the newly created rice variety exhibited both high yields and resistance to the fungus that causes a serious disease called rice blast.
Speaker: Simran Kaur, Co-founder & CEO at Tattva.Health
AI is transforming clinical trials—accelerating drug discovery, optimizing patient recruitment, and improving data analysis. But its impact goes far beyond research. As AI-driven innovation reshapes the clinical trial process, it’s also influencing broader healthcare trends, from personalized medicine to patient outcomes.
As clinical trials become increasingly complex, particularly in decentralized trials and rare disease studies, sponsors experience increased challenges in site selection, forecasting and resourcing, and patient recruitment and enrollment. Discover how AI is used to optimize key aspects of clinical trial management.
However, recent breakthroughs in AI, such as predictive modelling, clinical trial optimisation, and personalised medicine, have demonstrated its potential. We spoke with Aaron Smith, a mathematician-turned-machine learning scientist and the founder of Unlearn , a company leading the charge in applying AI to optimise clinical trial efficiency.
Researchers have achieved a significant breakthrough in identifying the primary cause and potential treatment for preeclampsia, a severe pregnancy complication. Upon testing this antibody in mice, the researchers found the all symptoms associated with preeclampsia were corrected.
A research team has now shown that inoculating the soil with mycorrhizal fungi can help maintain or even improve yields without the use of additional fertilizers or pesticides. In a large-scale field trial, plant yield increased by up to 40 percent. Farmland often harbors a multitude of pathogens which attack plants and reduce yields.
The conditional clearance for Sarepta’s Elevidys in 4- and 5-year-olds is a milestone for research into the deadly disease, and raises the stakes of an ongoing trial that could prove how well it works.
Science (2024) Related content New gene delivery vehicle shows promise for human brain gene therapy My Quest to Cure Prion Disease — Before It’s Too Late | Sonia Vallabh | TED Prion diseases lead to rapid neurodegeneration and death and are caused by misshapen versions of the prion protein in the brain.
By Makenzie Kohler October 23, 2023 People of South Asian ancestry around the world have more than double the risk of developing cardiometabolic diseases like diabetes, heart attack, and stroke compared to other populations. They will also have the opportunity to inform the researchers on whether their health status changes over time.
A medicine the pharma acquired in a $430 million buyout of Visterra in 2018 succeeded in a Phase 3 trial in IgA nephropathy, a crowded corner of drug research.
Earlier this year Pfizer removed thousands of participants from a study of its Lyme disease shot over concerns the group, Care Access, wasn't meeting clinical practice standards.
Food and Drug Administration (FDA) plays a pivotal role in fostering the development of treatments for rare diseases through its Orphan Products Grants Program. Each year, FDA selects a limited number of clinical trials to fund to help sponsors pursue development of medical products for rare diseases and advance their field.
28, 2023 -- In preliminary findings, Swedish researchers say taking a cholesterol-lowering statin could also slow the progression of Alzheimer's disease.The study can't prove cause-and-effect, but might pave the way to a trial that. THURSDAY, Dec.
PITTSBURGH – To reimagine existing preclinical trials for Alzheimer’s disease, University of Pittsburgh School of Medicine neuroscientists created the first non-human primate model of hereditary Alzheimer’s in marmoset monkeys, outlining their approach in Alzheimer’s & Dementia: Translational Research & Clinical Interventions.
Autoimmune diseases and bone health conditions are not just medical challenges – they’re personal battles that millions of people fight every day. Fortunately, advances in clinical research are providing hope for better treatments and outcomes.
While individual rare diseases affect populations that are small in numbers, collectively they impact millions globally, posing significant health and research challenges. Real-world data (RWD) and real-world evidence (RWE) have emerged as transformational tools in addressing these hurdles, advancing rare disease drug development.
The search for effective treatments for neurodegenerative diseases like Parkinson’s disease has long been hindered by the brain’s complexity and the absence of adequate models for drug discovery. Schwamborns research focuses on creating patient-specific brain organoids.
The figure depicts the neuroprotective role of crocin in Parkinson's disease. Abstract Parkinson's disease is among the most common forms of neurodegenerative illness, with present treatment being primarily symptomatic and frequently coming with substantial adverse effects.
Imagine being able to create an in vitro replica of a diseased organ to study the molecular mechanism underlying the illness. Now take a step further: envision testing drugs in these organoids to identify the ones that can treat disease safely and effectively without needing to run expensive clinical trials first.
Biosimilar clinical trials and study designs’ Considerations The rising prevalence and large market share of biological products emphasize their importance as treatment options for several cancers and autoimmune diseases.
Physicians working in the early 20th century had little choice but to treat the world’s most rampant infectious disease with methods such as these. Even after microbiologists discovered the bacterium that causes the illness in 1882, it wasn’t until the 1920s that researchers were able to develop a vaccine for TB.
For individuals living with a condition like melanoma, volunteering to participate in a clinical trial can be a rewarding experience. Individuals with Jamie’s specific diagnosis are given a 14% 5-year survival rate — but through participating in an immunotherapy clinical trial, Jamie is living without evidence of the disease.
Institutional review boards (IRBs) play a crucial role in the ever-evolving field of clinical research. Innovations in fields such as immunotherapy, cancer and chronic diseases owe their existence to clinical trials, which judiciously test and evaluate safety and efficacy.
In an era where clinical trials are increasingly global, it’s more imperative than ever to leverage international expertise. Data and safety monitoring boards (DSMBs), also known as data monitoring committees (DMCs), play a critical role in overseeing a clinical trial’s safety and efficacy.
Being the second most populous country in the world with skilled labor and good infrastructure, India is a favorable destination for clinical trials for a wide variety of diseases.
Having a one in four chance of developing sickle cell disease (SCD) is uniquely, and overwhelmingly, familiar to the African American population. Considered a rare disease, research in finding better therapies – and possibly even a cure – for SCD may be competing for funding with conditions impacting broader populations.
Pulmonary arterial hypertension (PAH) is a progressive and fatal lung disease that is caused or influenced by multiple factors. As a result, researchers now often use composite endpoints in lieu of the customary single primary endpoint. Historically, the available drugs and U.S. None offers a cure for PAH.
Rare diseases, therefore, present compelling opportunities for Drug Development. Since each disease is present in small groups of patients, this research is also fraught with complex challenges. Limited numbers of patients translate to scarce information, making this research risky and expensive. –5.9%
Autoimmune disorders comprise several diseases in which the immune system mistakenly attacks and destroys the body’s own healthy cells instead of destroying bacteria and viruses to keep the body healthy. […] The post Autoimmune disorders and current developments of novel treatments under clinical trials appeared first on ProRelix Research.
Importantly, the Hub is intended to establish a new model within FDA, which leverages cross-Agency expertise in providing guidance and conducting reviews for products for rare disease populations. Early Direction for the Rare Disease Innovation Hub The Hub, which will be co-chaired by Drs.
In the realm of vaccine development, mega trials — studies enrolling 5,000 subjects or more — have been instrumental in the fight against many pathogens, including influenza, rotavirus, malaria, RSV and most recently in the rapid development of vaccines against COVID-19.
The COVID-19 pandemic rapidly accelerated the adoption of hybrid and decentralized clinical trial (DCT) models. However, as the world settles into its post-pandemic state and returns to pre-pandemic paradigms in many areas, the pharmaceutical industry remains dedicated to moving beyond traditional, centralized clinical trial constructs.
Almost every one of us will know someone with a neurodegenerative disease such as Alzheimer’s or Parkinson’s. It is less common to know someone with a rare genetic disease; but while individually rare, collectively they have an enormous impact. This will, in turn, slow the progression of, or even halt, the disease.
What is Perthes’ disease? Perthes’ Disease is a rare condition that affects the developing femoral head (the “ball” of the “ball and socket” hip joint) of a child due to poor blood supply. This in turn can cause significant pain for children with Perthes’ disease and limit their activity levels significantly.
Adults aren’t the only patients affected by the increasing prevalence of liver disease. The number of pediatric patients diagnosed with nonalcoholic fatty liver disease (NAFLD) and nonalcoholic steatohepatitis (NASH) is on the rise, yet there are currently no approved therapies to treat NAFLD and NASH in adult or pediatric populations.
The rising prevalence of ophthalmology disorders such as dry eye disease (DED) and vision-related issues is largely related to the increasing use of mobile phones, tablet screens, and laptops.
Investment Trends in Pharmaceutical Research and Development Investing in the pharmaceutical sector presents a unique set of challenges and opportunities; let’s examine them in more detail. This exponential growth is primarily fueled by the escalating global burden of chronic diseases. trillion by 2028. billion by 2030.
Kidney diseases are one of the top causes of death in the United States , and worldwide, they impact nearly 10% of the population each year. Because chronic kidney disease typically has no cure, it is vital that medical research takes place to find new and more effective treatments for those who have been diagnosed.
By: Amy Raymond, Derek Ansel, Nathan Chadwick, & Juliane Mills When choosing a CRO for a rare disease study, what truly sets them apart is their methodology: the CRO’s mindset, their approach to each unique study, and their agility in navigating the inherent complexities of rare diseaseresearch.
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