Disease and Trials - Drug Discovery Digest

Diabetes Drug, Dapagliflozin, Effective For Fatty Liver Disease

Drugs.com

JUNE 6, 2025

FRIDAY, June 6, 2025 — A repurposed diabetes drug can be an effective tool against fatty liver disease, a new clinical trial has found. Dapagliflozin helped people reduce fat levels in their liver and avoid liver scarring that comes with the.

Disease

Disease Clinical Trials Drugs Trials

Combo Drug Treatment More Effective For Kidney Disease in Type 2 Diabetics

Drugs.com

JUNE 9, 2025

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.

Treatment

Treatment Disease Clinical Trials Drugs

Diabetes Drug Effective For Fatty Liver Disease

Drugs.com

JUNE 6, 2025

FRIDAY, June 6, 2025 — A repurposed diabetes drug can be an effective tool against fatty liver disease, a new clinical trial has found.Dapagliflozin helped people reduce fat levels in their liver and avoid liver scarring that comes with the c.

Disease

Disease Clinical Trials Drugs Trials

FDA lifts hold on PTC Huntington’s disease trial

BioPharma Drive: Drug Pricing

JUNE 20, 2024

The agency has lifted a partial trial suspension based on one-year data showing PTC’s pill suppressed a key protein associated with the disorder.

Trials

Trials Disease FDA

Bridging Innovation & Patient Care: The Growing Role of AI

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.

Clinical Trials

The AI model that is changing clinical trial design

Drug Target Review

JULY 1, 2025

Clinical trials are expensive, slow and often limited by outdated design constraints.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.

Clinical Trials

Clinical Trials Trials FDA Disease

Ozempic Can Treat Fatty Liver Disease

Drugs.com

MAY 1, 2025

THURSDAY, May 1, 2025 -- The cutting-edge weight-loss drug Ozempic/Wegovy can stop and even reverse fatty liver disease, a new clinical trial has found.Nearly twice as many people taking semaglutide stopped their fatty liver disease without further.

Disease

Disease Clinical Trials Trials Drugs

How the right technology and tools can accelerate progress in rare disease clinical trials

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.

Clinical Trials

Clinical Trials Disease Trials Research

Xenon gas could protect against Alzheimer's disease: Mouse study

Science Daily: Pharmacology News

JANUARY 15, 2025

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.

Disease

Disease Clinical Trials Treatment Trials

Novel bone marrow transplant can cure sickle cell disease, study suggests

Science Daily: Pharmacology News

FEBRUARY 25, 2025

A bone marrow transplant process is safe and curative for adults with sickle cell disease, according to results of a trial completed at about 20 cancer centers.

Disease

Disease Trials

New Antibiotic, Zoliflodacin, Seeking Approval, Tackles Drug-Resistant Gonorrhea in Trial

Drugs.com

NOVEMBER 3, 2023

3, 2023 -- The first new antibiotic for gonorrhea — the second most common sexually transmitted disease — has shown promise in a clinical trial. FRIDAY, Nov. That news should come as a relief to public health experts, because gonorrhea.

Trials

Trials Clinical Trials Disease Drugs

Merck antibody reduces RSV-related disease, hospitalizations in trial

BioPharma Drive: Drug Pricing

OCTOBER 18, 2024

Detailed data from a Phase 2b/3 study of Merck’s treatment clesrovimab should support the company’s efforts to win approval by the 2025-2026 season.

Hospitals

Hospitals Disease Trials Treatment

New tRNA tech aims to rewrite rare disease treatment

Drug Target Review

JUNE 9, 2025

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

Disease

Disease Treatment Engineering Protein Production

Discovery of 'new rules of the immune system' could improve treatment of inflammatory diseases, say scientists

Science Daily: Pharmacology News

JUNE 18, 2024

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.

Treatment

Treatment Immune Response Disease Clinical Trials

Experimental Drug Could Be Big Advance Against Kidney Disease

Drugs.com

DECEMBER 19, 2023

19, 2023 -- Early results from a trial of a new kidney disease medication show it significantly reduces levels of a urine marker of kidney damage.The experimental drug -- called BI 690517 for now -- cut levels of the liver protein. TUESDAY, Dec.

Disease

Disease Drugs Trials

More Evidence Gene Therapy Might Cure Sickle Cell Disease

Drugs.com

JUNE 12, 2023

MONDAY, June 12, 2023 -- A clinical trial that’s attempting to discover a cure for sickle cell disease has found a new gene therapy to be safe and successful in four patients. Two of the patients were treated at Cleveland Clinic Children’s in.

Therapies

Therapies Disease Clinical Trials Trials

Genome editing used to create disease resistant rice

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.

Disease

Disease Trials Research

Twice-a-Year Injection Gives Women Full Protection Against HIV, Trial Finds

Drugs.com

JUNE 24, 2024

MONDAY, June 24, 2024 -- Just two injections a year of a new HIV drug protected young women in Africa from infection with the sexually transmitted disease, new trial results show.In announcing the findings, Gilead Sciences Inc. said its HIV.

Trials

Trials Science Disease Drugs

ALS drug effectively treats Alzheimer's disease in new animal study

Science Daily: Pharmacology News

APRIL 8, 2025

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.

Small Molecule

Small Molecule Disease Clinical Trials Drugs

Leveraging AI Solutions for Clinical Trial Efficiencies

PPD

JANUARY 15, 2025

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.

Clinical Trials

Clinical Trials Trials Clinical Research Drug Development

Experimental GLP-1 Med Might Be Breakthrough Against Fatty Liver Disease

Drugs.com

JUNE 10, 2024

MONDAY, June 10, 2024 -- An experimental 'supercharged' form of popular GLP-1 weight-loss meds could help ease fatty liver disease, a new trial suggests. The drug under development, survodutide, helped up to 83% of patients gain real improvements.

Disease

Disease Trials Drugs

One Neurological Factor Keeps Black, Hispanic Patients From Alzheimer's Clinical Trials

Drugs.com

APRIL 19, 2024

FRIDAY, April 19, 2024 -- Black and Hispanic patients with Alzheimer’s disease are greatly underrepresented in clinical trials, even though they’re more likely to get dementia than whites.However, racial discrimination may not be driving this.

Clinical Trials

Clinical Trials Trials Disease

New data showcase promise, growing pains of CAR-T in autoimmune disease

BioPharma Drive: Drug Pricing

JUNE 17, 2024

One expert described trial results presented at EULAR last week as “unprecedented.” ” But reports of relapses in some patients drew questions about the therapies’ ultimate potential.

Disease

Disease Therapies Trials

10 clinical trials to watch in the first half of 2024

BioPharma Drive: Drug Pricing

JANUARY 2, 2024

A non-addictive pain pill faces its definitive test, while study results in ALS, a rare heart disease and lung cancer could have far-reaching implications.

Clinical Trials

Clinical Trials Trials Disease

When is a Confirmatory Trial “Underway” or Conducted with “Due Diligence” Enough for Accelerated Approval? FDA Explains Its New Authorities

FDA Law Blog: Drug Discovery

JANUARY 30, 2025

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.

FDA

FDA Trials Disease Drugs

CAR-T for lupus: the ‘tip of the iceberg’ for cell therapy in autoimmune disease

BioPharma Drive: Drug Pricing

JANUARY 30, 2024

Since a landmark paper in 2022, drugmakers have begun nearly a dozen trials of cell therapies for lupus, with more set to start. Here’s why their efforts are worth watching.

Therapies

Therapies Disease Trials

How AI will reshape pharma by 2025

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.

Clinical Trials

Clinical Trials Trials Pharmaceuticals Drug Development

'Exceptional' results in phase III leukemia trial

Science Daily: Pharmacology News

DECEMBER 10, 2023

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.

Trials

Trials Treatment Disease Drugs

Immunovant data show potential for autoimmune disease drug

BioPharma Drive: Drug Pricing

SEPTEMBER 26, 2023

Much anticipated clinical trial data for Immunovant’s experimental FcRn inhibitor could catalyze business development decisions for its parent, Roivant Sciences.

Clinical Trials

Clinical Trials Disease Science Trials

ASH23: CAR-T for autoimmune disease, real-world questions and sickle cell’s social media mentions

BioPharma Drive: Drug Pricing

DECEMBER 9, 2023

Study results disclosed at the American Society of Hematology meeting Saturday suggested potential for cell therapy in lupus and pointed out drug trial limitations.

Drug Trials

Drug Trials Disease Therapies Trials

3 ways decentralized clinical trials could help advance research on rare diseases

BioPharma Drive: Drug Pricing

NOVEMBER 13, 2023

Explore how decentralized clinical trials (DCTs) can enhance rare disease research in three key ways.

Clinical Trials

Clinical Trials Trials Disease Research

ASH23: Pharma branding, Editas’ high bar and clinical trial diversity

BioPharma Drive: Drug Pricing

DECEMBER 11, 2023

Editas had the tall task Monday of convincing ASH attendees its gene therapy for sickle cell disease could improve on Casgevy and Lyfgenia.

Clinical Trials

Clinical Trials Trials Therapies Disease

Obesity drug from Boehringer, Zealand succeeds in MASH trial

BioPharma Drive: Drug Pricing

FEBRUARY 26, 2024

Zealand shares rose by more than one-third on the data, which provide further evidence so-called incretin drugs could help treat people with the liver disease.

Trials

Trials Drugs Disease

After surprise trial failure, ALS doctors brace for one less treatment option

BioPharma Drive: Drug Pricing

MARCH 12, 2024

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.

Doctors

Doctors Treatment Trials Pharmaceuticals

Wegovy Cuts Heart Risks by 20% in Large Trial

Drugs.com

NOVEMBER 14, 2023

13, 2023 (Healthday News) -- In a finding that could change the landscape of heart disease care, the wildly popular weight-loss drug Wegovy has proved its mettle in protecting the heart after lowering the risk of cardiac problems in. MONDAY, Nov.

Trials

Trials Disease Drugs

10 clinical trials to watch in the second half of 2023

BioPharma Drive: Drug Pricing

JUNE 27, 2023

A Duchenne gene therapy faces a crucial test, while highly anticipated study results are expected in lung cancer, obesity and heart disease.

Clinical Trials

Clinical Trials Trials Therapies Disease

Inoculation against diseased fields

Science Daily: Pharmacology News

NOVEMBER 30, 2023

In a large-scale field trial, plant yield increased by up to 40 percent. 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.

Disease

Disease Trials Research

The Growing Trend of Patient-Led Clinical Research in Rare Disease

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?

Clinical Research

Clinical Research Disease Research Clinical Trials

Verve gets FDA green light to run base editing study in US

BioPharma Drive: Drug Pricing

OCTOBER 23, 2023

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.

FDA

FDA Trials Treatment Disease

Roche autoimmune disease drug disappoints in closely watched trial

BioPharma Drive: Drug Pricing

MARCH 21, 2024

Though the medicine hit its goal in a Phase 3 study in myasthenia gravis, the results fell short of expectations in a boost to rival drug developers like Argenx and AstraZeneca.

Disease

Disease Trials Drugs Drug Development

Anti-amyloid drug shows signs of preventing Alzheimer's dementia

Science Daily: Pharmacology News

MARCH 19, 2025

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.

Clinical Trials

Clinical Trials Drugs Trials Treatment

Accelerate Clinical Trials with Methylation-Based ctDNA Detection

Fierce BioTech

JUNE 24, 2025

Accelerate Clinical Trials with Methylation-Based ctDNA Detection Download now to learn from biopharma industry leaders about how methylation-based ctDNA detection for precise MRD and response monitoring can accelerate your clinical trials.

Clinical Trials

Clinical Trials Trials DNA Disease

Ozempic Can Prevent Heart Trouble in Folks With Kidney Issues

Drugs.com

SEPTEMBER 12, 2024

12, 2024 -- Ozempic and Wegovy can prevent heart problems in overweight and obese people, particularly if they also suffer from kidney disease, a new clinical trial shows.The drugs’ active ingredient, semaglutide, reduced heart h. THURSDAY, Sept.

Clinical Trials

Clinical Trials Trials Disease Drugs

Chimeric deubiquitinase engineering reveals structural basis for specific inhibition of the mitophagy regulator USP30

Covalent Modifiers

MAY 18, 2025

Whether enhanced mitochondrial quality control through inhibition of USP30 can protect dopaminergic neurons is currently being explored in a clinical trial for Parkinsons disease. However, the molecular basis for specific inhibition of USP30 by small molecules has remained elusive.

Engineering

Engineering Regulations Small Molecule Clinical Trials

Diabetes Drug, Dapagliflozin, Effective For Fatty Liver Disease

Combo Drug Treatment More Effective For Kidney Disease in Type 2 Diabetics

Trending Sources

Diabetes Drug Effective For Fatty Liver Disease

FDA lifts hold on PTC Huntington’s disease trial

Bridging Innovation & Patient Care: The Growing Role of AI

The AI model that is changing clinical trial design

Ozempic Can Treat Fatty Liver Disease

How the right technology and tools can accelerate progress in rare disease clinical trials

Xenon gas could protect against Alzheimer's disease: Mouse study

Novel bone marrow transplant can cure sickle cell disease, study suggests

New Antibiotic, Zoliflodacin, Seeking Approval, Tackles Drug-Resistant Gonorrhea in Trial

Merck antibody reduces RSV-related disease, hospitalizations in trial

New tRNA tech aims to rewrite rare disease treatment

Discovery of 'new rules of the immune system' could improve treatment of inflammatory diseases, say scientists

Experimental Drug Could Be Big Advance Against Kidney Disease

More Evidence Gene Therapy Might Cure Sickle Cell Disease

Genome editing used to create disease resistant rice

Twice-a-Year Injection Gives Women Full Protection Against HIV, Trial Finds

ALS drug effectively treats Alzheimer's disease in new animal study

Leveraging AI Solutions for Clinical Trial Efficiencies

Experimental GLP-1 Med Might Be Breakthrough Against Fatty Liver Disease

One Neurological Factor Keeps Black, Hispanic Patients From Alzheimer's Clinical Trials

New data showcase promise, growing pains of CAR-T in autoimmune disease

10 clinical trials to watch in the first half of 2024

When is a Confirmatory Trial “Underway” or Conducted with “Due Diligence” Enough for Accelerated Approval? FDA Explains Its New Authorities

CAR-T for lupus: the ‘tip of the iceberg’ for cell therapy in autoimmune disease

How AI will reshape pharma by 2025

'Exceptional' results in phase III leukemia trial

Immunovant data show potential for autoimmune disease drug

ASH23: CAR-T for autoimmune disease, real-world questions and sickle cell’s social media mentions

3 ways decentralized clinical trials could help advance research on rare diseases

ASH23: Pharma branding, Editas’ high bar and clinical trial diversity

Obesity drug from Boehringer, Zealand succeeds in MASH trial

After surprise trial failure, ALS doctors brace for one less treatment option

Wegovy Cuts Heart Risks by 20% in Large Trial

10 clinical trials to watch in the second half of 2023

Inoculation against diseased fields

The Growing Trend of Patient-Led Clinical Research in Rare Disease

Verve gets FDA green light to run base editing study in US

Roche autoimmune disease drug disappoints in closely watched trial

Anti-amyloid drug shows signs of preventing Alzheimer's dementia

Accelerate Clinical Trials with Methylation-Based ctDNA Detection

Ozempic Can Prevent Heart Trouble in Folks With Kidney Issues

Chimeric deubiquitinase engineering reveals structural basis for specific inhibition of the mitophagy regulator USP30

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