On Medicine

FEBRUARY 28, 2024

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.

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Disease Doctors Treatment Trials 109

BioPharma Drive: Drug Pricing

AUGUST 7, 2023

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.

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Clinical Trials Disease Trials Research 87

Science Daily: Pharmacology News

JUNE 18, 2024

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.

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Treatment Disease Immune Response Clinical Trials 129

ProRelix Research

JUNE 6, 2023

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.

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Clinical Trials Trials Drugs Disease 98

Advarra

MARCH 10, 2023

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.

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PPD

AUGUST 5, 2024

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.

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Clinical Trials Trials Clinical Research Disease 98

Drug Target Review

DECEMBER 20, 2024

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.

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Broad Institute

NOVEMBER 21, 2024

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.

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Research Medical Schools DNA Hospitals 124

ProRelix Research

SEPTEMBER 7, 2023

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.

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Clinical Trials Trials Treatment Virus 105

FDA Law Blog: Drug Discovery

JULY 17, 2024

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.

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Drug Target Review

OCTOBER 11, 2023

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.

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PPD

AUGUST 16, 2023

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.

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Clinical Trials Trials Clinical Research FDA 97

PPD

JUNE 24, 2024

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.

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Science Daily: Pharmacology News

JUNE 15, 2023

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.

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Drug Target Review

AUGUST 28, 2024

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.

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Therapies Disease Engineering Medical Schools 116

Conversations in Drug Development Trends

OCTOBER 29, 2024

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 Disease Research?

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PPD

JUNE 7, 2023

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.

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Trials Clinical Trials Clinical Research Disease 98

ProRelix Research

SEPTEMBER 17, 2023

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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Clinical Trials Trials Treatment Disease 98

Antidote

JULY 27, 2022

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.

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Broad Institute

OCTOBER 11, 2023

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.

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Conversations in Drug Development Trends

MAY 14, 2024

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 disease research.

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ASPET

DECEMBER 22, 2023

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).

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Conversations in Drug Development Trends

AUGUST 23, 2023

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.

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SCIENMAG: Medicine & Health

SEPTEMBER 7, 2023

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.

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Drug Target Review

NOVEMBER 29, 2023

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.

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Therapies Disease RNA Engineering 102

Cytel

MAY 14, 2024

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.

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Clinical Trials Disease Trials Clinical Research 59

PPD

JULY 29, 2024

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.

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Conversations in Drug Development Trends

MARCH 1, 2024

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?

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Trials Disease Drug Development Clinical Trials 78

Drug Target Review

JUNE 21, 2023

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.

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Disease Clinical Trials Therapies Science 111

Advarra

AUGUST 8, 2024

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.

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PPD

OCTOBER 9, 2023

“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.

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Clinical Trials Trials Compliance International 98

Broad Institute

NOVEMBER 2, 2023

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.

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DrugBank

JULY 20, 2023

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%

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SCIENMAG: Medicine & Health

JULY 17, 2023

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 […]

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PPD

MAY 18, 2023

For neurodegenerative disease research, 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.

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Clinical Trials Trials Clinical Research Doctors 74

DrugBank

AUGUST 9, 2024

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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FDA Law Blog: Drug Discovery

MARCH 13, 2023

Who better than people living with a condition to inform drug companies, physicians, academics, and the FDA on what it is like to live with their condition, what symptoms most impact their lives, what goes into their decision about whether to participate in a clinical trial, and what kind of treatment effects would be most meaningful to them?

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