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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Clinical Trials Trials Pharmaceuticals Drug Development 128

Science Daily: Pharmacology News

SEPTEMBER 15, 2023

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.

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Science Daily: Pharmacology News

NOVEMBER 30, 2023

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.

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Disease Trials Research 226

Science Daily: Pharmacology News

JULY 15, 2024

Early exposure to antibiotics can trigger long term susceptibility to asthma, according to new research.

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Trials Treatment Disease Research 276

Broad Institute

OCTOBER 23, 2023

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.

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Broad Institute

APRIL 5, 2025

The organization has honored Liu for the development of the gene editing platforms base editing and prime editing, which can correct the vast majority of known disease-causing genetic variations and have already been used in at least 15 clinical trials, with life-saving results.

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Science Clinical Trials DNA Disease 139

BioPharma Drive: Drug Pricing

OCTOBER 22, 2024

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.

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Disease Drugs Drug Research Trials 167

FDA Law Blog: Drug Discovery

NOVEMBER 10, 2024

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.

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Drugs.com

DECEMBER 28, 2023

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.

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SCIENMAG: Medicine & Health

SEPTEMBER 6, 2023

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.

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Conversations in Drug Development Trends

OCTOBER 14, 2024

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.

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Conversations in Drug Development Trends

JANUARY 24, 2024

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.

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Drug Target Review

MARCH 3, 2025

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.

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Disease Treatment Therapies Cell Biology 52

Chemical Biology and Drug Design

MAY 11, 2023

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.

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Drug Target Review

OCTOBER 30, 2024

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.

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Treatment Disease Clinical Trials Molecular Biology 106

ProRelix Research

JULY 6, 2023

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.

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Clinical Trials Biosimilars Trials Treatment 105

Antidote

JANUARY 29, 2024

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.

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Clinical Trials Trials Doctors Treatment 122

Conversations in Drug Development Trends

NOVEMBER 5, 2024

In preparation for World Orphan Drug Congress Europe, we interviewed Nathan Chadwick, Senior Director, Therapeutic Strategy Lead, Rare Disease, and Derek Ansel, MS, LCGC, Vice President, Therapeutic Strategy Lead, Rare Disease, to hear their insights into the current progress in rare disease research and their hopes for 2025.

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Disease Clinical Trials Therapies Clinical Research 52

Drug Target Review

APRIL 7, 2025

As our understanding of the underlying biology of disease grows more sophisticated, emerging therapies operate on increasingly complex biopathological systems and mechanisms. A surrogate endpoint is a marker used in clinical trials as a substitute for a direct clinical outcome.

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Clinical Development Clinical Trials Clinical Pharmacology Trials 59

Advarra

NOVEMBER 14, 2024

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.

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PPD

MARCH 11, 2025

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.

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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

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

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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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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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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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 98

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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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

Advarra

MARCH 6, 2025

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.

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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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Olympian Clinical Research

AUGUST 5, 2020

Hidradenitis Suppurativa is a skin condition, not an infectious disease. . Researchers are working every day to develop a cure for HS, and they’re making great strides when it comes to treatments. The post A Closer Look At Hidradenitis Suppurativa: Disease, Causes, & Treatment appeared first on Olympian Clinical Research.

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SCIENMAG: Medicine & Health

JUNE 22, 2023

The UK is to launch a clinical trial of a ‘poo transplant’ that researchers believe could treat advanced liver disease and fight antimicrobial resistance. The trial’s investigators also provide evidence for the first time that a faecal transplant can dramatically improve gut health.

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LifeSciVC

JULY 17, 2024

By Mike Cloonan, Chief Executive Officer of Sionna Therapeutics, as part of the From The Trenches feature of LifeSciVC The drug development process in rare diseases is rife with challenges especially when companies target significant differentiation or first-in-class targets.

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