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MONDAY, June 9, 2025 — Prompt treatment with a drug combo can effectively manage chronic kidney disease in people with type 2 diabetes, a new clinical trial shows. Patients prescribed the combination of finerenone and empagliflozin.
The landscape of genetic medicine is undergoing a profound transformation, driven by innovative approaches that challenge the traditional, disease-specific paradigms. My transition to biotech and rare diseases was deeply personal – my son was diagnosed with Duchenne muscular dystrophy in 2020. “My
Clinical trials are expensive, slow and often limited by outdated design constraints. Placebo arms, in particular, create ethical and logistical hurdles, especially in areas like rare disease and oncology. At Unlearn, he is focused on bringing digital twin technology into active use across both early and late-phase trials.
The discovery could lead to more targeted treatments, with fewer side-effects, for issues from lengthy COVID infections to autoimmune diseases like multiple sclerosis. Clinical trials in humans are now planned.
Speaker: Simran Kaur, Co-founder & CEO at Tattva.Health
AI is transforming clinical trials—accelerating drug discovery, optimizing patient recruitment, and improving data analysis. As AI-driven innovation reshapes the clinical trial process, it’s also influencing broader healthcare trends, from personalized medicine to patient outcomes. But its impact goes far beyond research.
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.
Physicians are preparing for tough conversations with patients on Amylyx Pharmaceuticals’ drug, Relyvrio, while holding out hope it still may help some with the disease.
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.
Food and Drug Administration (FDA) for clinical trials for the treatment of amyotrophic lateral sclerosis (ALS) -- improves neuron health in animal models of Alzheimer's disease, according to a new study. Experimental drug NU-9 -- a small molecule compound approved by the U.S.
Leukaemia trial shows 'exceptional' results. Personalized treatment for the most common form of adult leukemia helps patients survive for longer and stay in remission, a phase III trial has found.
However, as we note in that post, the design, timing of initiation, and timely conduct of confirmatory trials are also important considerations in FDAs determination of whether accelerated approval is appropriate. This blog post focuses on interpreting these new authorities with respect to timely conduct of confirmatory trials.
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.
The trial, which is ongoing in the U.K. since late last year as the FDA sought more details on Verve’s in vivo treatment for heart disease. and New Zealand, has been on hold in the U.S.
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?
The study, of Intellia’s treatment for hereditary angioedema, is the second Phase 3 trial the biotech has begun testing its “in vivo” gene editing medicines.
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. Most of these conditions are genetic in origin and the majority have no effective treatment.
Liquid biopsy has emerged as a revolutionary tool for clinical trials, offering cellular and cell-free methods that provide valuable insights into disease progression and treatment response.
An experimental drug appears to reduce the risk of Alzheimer's-related dementia in people destined to develop the disease in their 30s, 40s or 50s, according to the results of a new study.
According to our analysis of clinical trial listings, the following industries are leading the charge in seeking legal counsel: Biotech: With a 25% increase in clinical trial listings over the past year, biotech companies are racing to secure patents and protect their intellectual property.
TUESDAY, June 18, 2024 -- There's more good news in the battle against sickle cell disease, with another trial finding CRISPR gene-editing therapy delivering impressive results for patients.“It’s It’s encouraging that this gene-editing treatment con.
Patients diagnosed with idiopathic pulmonary fibrosis (IPF) — a progressive, rare lung disease marked by scarring and worsening lung function — often face a poor prognosis. While two approved treatments, pirfenidone and nintedanib, slow disease progression, there currently is no treatment that reverses the effects or offers a cure for IPF.
Biogen Reports Promising Interim Phase 1 Results for Salanersen in Spinal Muscular Atrophy, Prepares for Registrational Trials Biogen has announced encouraging topline results from its Phase 1 clinical trial evaluating salanersen (BIIB115/ION306) , an investigational antisense oligonucleotide (ASO) therapy for spinal muscular atrophy (SMA).
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.
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.
These findings represent a pivotal advancement in the treatment of this rare, progressive, and life-threatening disease. Participants were classified as WHO Group 1 PAH, functional class (FC) II or III, indicating moderate to severe disease at either intermediate or high risk of clinical deterioration.
With 20-25% of the global population affected by metabolic problems, finding effective treatments is crucial for cardiovascular and metabolic (CVM) conditions. Traditional study designs feature predetermined sample sizes, assessments, and analyses that must remain in place over long treatment durations.
The IDeate-Prostate01 trial marks a significant advancement in the B7-H3 ADC development program and underscores the shared commitment of both companies to addressing difficult-to-treat cancers. This has created an urgent need for more effective and tolerable treatments for this population. There is a critical need for new treatments.
Importantly the research team isolated a molecule produced by gut bacteria that in the future could potentially be trialed as a simple treatment, in the form of a dietary supplement, for children at risk of asthma to prevent them developing the disease.
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.
A large-scale clinical trial of treatment strategies for Crohn's disease has shown that offering early advanced therapy to all patients straight after diagnosis can drastically improve outcomes, including by reducing the number of people requiring urgent abdominal surgery for treatment of their disease by ten-fold.
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.
Biogen Launches Global Phase 3 PROMINENT Trial Evaluating Felzartamab in Primary Membranous Nephropathy Biogen Inc. The trial is expected to complete in 2029. By selectively depleting these cells, felzartamab offers a promising, mechanism-driven approach to potentially halting disease progression.
The results come from a open-label, proof-of-concept, Phase 2 study (NCT04520451) and highlight rilzabrutinib’s potential as a disease-changing treatment option for a condition that currently has limited and non-specific treatment options and involves substantial patient suffering due to its chronic and progressive course.
A randomised controlled clinical trial is the most prevalent design used in Phase II and Phase III clinical trials, whereby participants are randomly assigned to a control or experimental group with a fixed probability, normally 50% for each treatment.
The study also explores and compiles the latest patents and clinical trial data into a single platform to help develop selective Mtb -DHFR inhibitors for effective tuberculosis treatment. ABSTRACT Tuberculosis (TB) is an obstinate and infectious disease requiring a relatively longer treatment duration than other bacterial infections.
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.
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. “For diseases like Parkinson’s, it’s more than sufficient,” he explains.
Dapirolizumab Pegol Demonstrates Promise in Phase 3 SLE Trial, Showing Improvements in Fatigue and Disease Activity at EULAR 2025 UCB (Euronext Brussels: UCB) and Biogen Inc. The latest data from the PHOENYCS GO trial reinforce the potential of dapirolizumab pegol to improve disease outcomes in people with moderate-to-severe SLE.
The drug, which is already cleared as a neoadjuvant lung cancer treatment, could further compete with Merck’s Keytruda if it gains approval in the adjuvant setting for patients with the non-small cell form of the disease.
LRRK2 has been a hotly pursued drug target for Parkinson’s Disease based on human genetics. On Sunday, it was disclosed that additional, likely on-target adverse findings were observed in a primate GLP study on long-term treatment with Merck’s second-generation LRRK2 inhibitors. Get ahead now by requesting a trial.
Ionis Begins Pivotal Phase 3 REVEAL Study of ION582 in Angelman Syndrome, Dosing First Patient in Global Trial Ionis Pharmaceuticals , Inc. Currently, treatment options are limited to supportive care aimed at managing symptoms rather than targeting the underlying cause.
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