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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.
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. One diagnostic example that I discussed in my presentation is autism.
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.
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.
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.
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.
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.
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.
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.
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.
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 identify source of a brain cancer’s deadly transformation By Corie Lok November 21, 2024 Breadcrumb Home Researchers identify source of a brain cancer’s deadly transformation Slow-growing glioma tumors become more aggressive when their cells shift identities and acquire new genetic mutations.
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.
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.
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.
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.
What are the main challenges currently faced in the treatment of chronic liver diseases, and how does Resolution Therapeutics aim to address these challenges? Once a patient develops advanced cirrhosis/end-stage liver disease there are no specific therapies to significantly avoid major decompensations and death in the next few years.
By: Juliane Mills, Senior Director, Therapeutic Strategy Lead, Rare Disease The rise of patient-led clinical research, particularly in rare disease, represents a significant shift in the clinical trial landscape. Why Is There an Increase in Patient-Led Rare DiseaseResearch?
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.
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.
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Manguso, who’d recently graduated from college and was conducting research at the University of Copenhagen as a Fulbright scholar, moved back to the Boston area to be with his mother as she underwent treatment. His mother had a presentation of the disease that suggested her immune system was already on the job.
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.
This study provides a unique translational research opportunity to help both humans and dogs diagnosed with diseases that carry dismal prognoses in both species: histiocytic sarcoma (HS), hemangiosarcoma (HSA), and disseminated mastocytosis/mast cell tumor (MCT).
Patients are the backbone of clinical trials, playing an essential role in the drug development process. This engagement is often less understood and is underutilized by sponsors, meaning a significant element of the trial and drug experience is missed during sponsor engagement with the FDA.
7, 2023 – Alzheimer’s disease is the most common cause of dementia that affects more than 6.5 To find effective treatments and slow the progression of this debilitating disease, researchers have made much progress in developing new drugs that target beta-amyloid plaques, one of […] WINSTON-SALEM, N.C.
Our primary focus is to design and develop RNA therapies for liver diseases. Using humans as the model, we use an approach called deep phenotyping to explore the relationships between cells, genes, biological pathways and patterns of disease. Why are you focusing on liver disease? As a result, waiting lists continue to grow.
Written by Angela Vinken and Patti Arsenault Key Opinion Leaders (KOLs) — i.e., trusted, well-respected experts — are crucial in clinical research, especially in rare diseases. The challenge, however, is how to use KOLs wisely — they are often clinicians first and researchers second, and their time is in high demand.
AI also has the potential to incorporate real-world data (RWD) obtained from electronic health records (EHRs), medical claims or other data sources to inform the design and optimization strategy of clinical trials. A high-risk participant can be even excluded from the study based on the severity of the adverse event.
These designs facilitate streamlined trial logistics and centralized governance and create higher-quality data. Master protocols allow a trial to perform multiple tests on diverse patient populations or diseases under a unified design. But how do they work, and when are they appropriate? What Are Master Protocols?
Most rare diseases are caused by a single gene defect, but severity can vary considerably among patients. Modifier genes can help explain that variability and can alter or even prevent disease onset and progression, making them appealing therapeutic targets. However, the identification of these genes is challenging.
The EU AI Act’s implications extend into clinical research, where AI is increasingly utilized for tasks like medical image analysis, natural language process for endpoint analysis, and generating/analyzing data for synthetic control arms. Here are some key requirements for “high risk” AI systems as they relate to clinical trials.
“Patient-centric” is used frequently to describe today’s clinical trial and health care landscape. The industry applauds that point of view and strives to include a patient-centric approach in all aspects of clinical trial designs. Education on the disease state and regular reinforcement.
Bayer and Broad Institute extend cancer therapy research collaboration By Corie Lok November 2, 2023 Breadcrumb Home Bayer and Broad Institute extend cancer therapy research collaboration Long-standing industry-academia collaboration has already resulted in three clinical oncology candidates.
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%
Osaka, Japan – Systemic autoimmune diseases are characterized by inflammation of multiple organs and can have devastating consequences for patients. There is a dire need for treatments against these diseases. RNase treatments seem promising in some clinical trials but not all. Credit: Ryota Naito et […]
For neurodegenerative diseaseresearch, the care partner, or caregiver, is equally important as the person with dementia. Even with stronger awareness of studies, drug developers must contend with the inherent lack of trust between the dementia community and clinical trials. There’s little time to lose.
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.
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.
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