Drug Target Review

APRIL 7, 2025

Haemoglobin A1c (HbA1c) is a validated surrogate endpoint for the reduction of microvascular complications associated with diabetes mellitus; reduced HIV-RNA levels serve as an endpoint for HIV disease control; and a reduction in low-density lipoprotein (LDL) cholesterol is used as an endpoint indicating lower likelihood of cardiovascular events.

Clinical Development 59

Clinical Development Clinical Trials Clinical Pharmacology Trials 59

Covalent Modifiers

SEPTEMBER 19, 2023

No dose-limiting toxic effects or treatment-related deaths were reported. Treatment-related adverse events occurred in 127 patients (93%); grade 3 events occurred in 15 patients (11%) and a grade 4 event in 1 patient (1%). Among patients with NSCLC, a confirmed response was observed in 53.4% months (95% CI, 8.8

Pharmacokinetics 147

Pharmacokinetics DNA Treatment 147

SCIENMAG: Medicine & Health

JUNE 24, 2023

About The Study: The findings of this study suggest that treatment with bempedoic acid in high-risk primary prevention patients unable to tolerate recommended doses of statins has the potential to reduce major adverse cardiovascular events. Authors: Steven E. Nissen, M.D., of the Cleveland Clinic, is the corresponding author.

Treatment 68

Treatment 68

PLOS: DNA Science

MARCH 28, 2024

FDA classifies it as a “nonsteroidal treatment” – not a gene therapy, but it affects gene expression. A gene-based treatment would have to alter many cells to exert a noticeable effect. In 2023, two gene-based treatments became available. Muscle makes up about 40 percent of body weight. million DNA bases.

FDA Approval 97

FDA Approval Treatment FDA DNA 97

Chemical Biology and Drug Design

FEBRUARY 13, 2024

Some researchers proposed that the symptoms involved in AD (loss of memory, cognitive impairment, and amnesia) are the main event linked to the cholinergic neurotransmitter (acetylcholine). This review focuses on the last 10 year's literature search on huprine and its analogues for the treatment of AD.

Disease 100

Disease Treatment Therapies Research 100

Broad Institute

FEBRUARY 25, 2025

While more than 8,000 genes are known to drive these diseases, fewer than 500 have an available treatment. The Ladders to Cures Scientific Accelerator is committed to uncovering the genetic roots of rare diseases and to using those insights to develop new treatments.

Disease 52

Disease Treatment 52

ASPET

SEPTEMBER 22, 2023

These results suggest that brain damage can be long-lasting or permanent if nerve agent-induced SE in infant victims is treated with midazolam at a delayed timepoint after SE onset, while antiglutamatergic treatment with tezampanel and caramiphen provide significant neuroprotection.

Treatment 100

Treatment FDA Drugs 100

Broad Institute

JULY 11, 2024

Targeted drug treatment leads tumor cells to imitate viral infection By Ari Navetta July 11, 2024 Breadcrumb Home Targeted drug treatment leads tumor cells to imitate viral infection Exploiting "viral mimicry," mIDH1 inhibitors trick tumors into thinking they are infected with a virus.

Treatment 72

Treatment Immune Response Drugs Virus 72

Broad Institute

NOVEMBER 25, 2024

Ladders to Cures (L2C) Accelerator By Maria Nemchuk November 25, 2024 Breadcrumb Home Ladders to Cures (L2C) Accelerator The Ladders to Cures (L2C) Accelerator aims to catalyze progress across the research ecosystem and accelerates advances leading to treatments and cures for patients with rare genetic diseases. Visit broad.io/L2C2023

Clinical Trials 52

Clinical Trials Disease Trials Therapies 52

Elrig

MARCH 25, 2025

Prof Rory Johnson, Associate Professor, University College Dublin, and Dr Shalini Andersson, Vice President Nucleic Acid Therapeutics, AstraZeneca will lead this years event focussed on drugging the undruggable.

RNA 59

RNA Bioinformatics Science Pharmacokinetics 59

KIF1A

JULY 15, 2023

When the brain’s electrical activity changes enough to cause noticeable changes in behavior, we call that event a seizure. By identifying short and subtle changes in movements, we may be able to characterize epileptic events with more precision. From Gschwind et al. 2023, Neuron.

Disease 98

Disease Treatment Research Therapies 98

DrugBank

OCTOBER 17, 2024

Bayesian adaptive randomization , for example, can dynamically allocate patients to different treatment arms based on accumulating data, ensuring that more patients receive the most promising treatment. Machine learning algorithms can be trained on historical data to identify patterns and anomalies indicating potential safety concerns.

Drug Development 98

Drug Development Metabolic Stability Clinical Trials Drugs 98

PPD

AUGUST 7, 2024

Clinical trials are the backbone of medical research, enabling the development of new treatments and therapies that can improve patient outcomes. Adverse Event Reporting and Safety Monitoring: During a clinical trial, participants may experience adverse events that may be related or unrelated to the study drug.

Clinical Trials 97

Clinical Trials Trials Compliance Clinical Research 97

Drug Target Review

AUGUST 6, 2024

Autoimmune diseases : Rheumatoid Arthritis : ADCs targeting specific immune cells or inflammatory mediators can provide more precise treatment options with potentially fewer side effects. Obesity : By targeting adipose tissue or specific metabolic pathways, ADCs could offer new treatments for obesity and related metabolic disorders.

Therapies 119

Therapies Pharmaceutical Companies Disease Treatment 119

ASPET

JUNE 30, 2023

Myeloproliferative Neoplasms (MPNs) are hematological malignancies that result from acquired driver mutations in hematopoietic stem cells (HSCs), causing overproduction of blood cells and an increased risk of thrombo-hemorrhagic events. This study aims to determine a personalized treatment strategy.

Therapies 100

Therapies Treatment Drugs 100

SCIENMAG: Medicine & Health

JANUARY 16, 2024

Scaling drugs to a patient’s weight prevents adverse events from overtreatment and treatment failure due to underdosing. Knowing a patient’s weight is necessary for many weight-based medications such as thrombolytics, anticoagulants and numerous cardiovascular medications. Credit: Alex Dolce, Florida Atlantic University […]

Research 89

Research Treatment Drugs 89

Chemical Biology and Drug Design

MAY 11, 2023

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. Therefore, this compound is considered a safe and effective therapeutic choice for neurodegenerative illnesses like Parkinson's disease.

Disease 100

Disease Research Clinical Trials Treatment 100

Broad Institute

JUNE 28, 2023

The findings, published today in Nature , could lead to new ways of guiding cancer treatment or developing targeted drugs. We began to think that these shorter events could give us signals about whether cancer cells were selecting for certain chromosome changes.”

DNA 122

DNA Medical Schools Treatment Research 122

FDA Law Blog: Biosimilars

JUNE 12, 2024

Gibbs — It’s been over a year and a half since Over-the-Counter (“OTC”) hearing aids became legal, and it’s not clear that they’ve made the difference in hearing loss treatment that Congress anticipated. (FDA FDA does, however, expect to issue a report on OTC hearing aid adverse events to Congress by August 2024.

FDA 111

FDA Regulations Treatment Licensing 111

The Pharma Data

MARCH 28, 2022

mg dose of Ozempic ® (once-weekly semaglutide subcutaneous injection), a glucagon-like peptide-1 (GLP-1) analogue for the treatment of adults with type 2 diabetes. mg doses for the treatment of type 2 diabetes in adults. The most common adverse events were gastrointestinal. Ozempic ® is now approved in the US at 0.5 mg and 2.0

Treatment 52

Treatment FDA Approval Trials FDA 52

FDA Law Blog: Drug Discovery

JANUARY 30, 2025

This target completion date is informed by the natural history of the disease, availability of alternative treatment, anticipated recruitment timeline, and the projected timeline for efficacy analysis(es); 2) the sponsors progress and plans for postapproval conduct of the trial provide sufficient assurance to expect timely completion of the trial.

FDA 105

FDA Trials Disease Drugs 105

The Pharma Data

MAY 16, 2022

Food and Drug Administration (FDA) has lifted the clinical hold placed on the company’s Investigational New Drug Application (IND) to evaluate injectable lenacapavir for HIV treatment and HIV pre-exposure prophylaxis (PrEP). There is no cure for HIV or AIDS. About Lenacapavir.

Treatment 52

Treatment FDA Science Therapies 52

SCIENMAG: Medicine & Health

NOVEMBER 12, 2023

Low levels of Vitamin D have been shown to be associated with a higher risk of having a cardiac event, like a heart attack or stroke. For this reason, treatment by Vitamin D pills or injections are being investigated as a possible preventative method in these patients. Credit: Intermountain Health Low levels of Vitamin D […]

Treatment 83

Treatment 83

Conversations in Drug Development Trends

AUGUST 10, 2023

As one of the more recent fields to emerge in the clinical space, psychedelics have gained significant traction as a potential treatment for various neuropsychiatric diseases. I was fortunate to have the opportunity to attend Psychedelic Science 2023 during the third week of June in Denver, Colorado.

Science 98

Science Clinical Trials Trials Clinical Research 98

PPD

JULY 29, 2024

The benefits of utilizing AI in clinical research Patient recruitment and diversity AI is increasingly being utilized in the recruitment process for clinical trials , aiming to connect individuals with potential benefits from investigational treatments.

Clinical Research 98

Clinical Research Clinical Trials Research Trials 98

Eye on FDA

JULY 29, 2021

Perhaps no other decision by the Food and Drug Administration has garnered as much controversy, as the recent one to authorize the accelerated approval a new treatment for Alzheimer’s. Fundamental to any crisis response is the development and delivery of a narrative that offers explanation for the events. They are: What happened?

FDA 105

FDA Vaccine Clinical Trials Treatment 105

Drug Target Review

JULY 4, 2023

These therapies have broadened treatment options for patients to expand beyond the more traditional small molecule drug alternatives. ADCs have the potential to redress the poor balance between safety and efficacy seen with traditional cancer treatment options.

Small Molecule 145

Small Molecule Drugs Clinical Pharmacology Trials 145

Alta Sciences

NOVEMBER 4, 2024

We anticipated potential hazards and recognized and evaluated all potential exposure events to mitigate risk. Identifying exposure events early on—particularly for the dispensing of the API into the vehicle—enabled our experts to implement appropriate containment measures.

Engineering 52

Engineering Production Compliance Laboratories 52

Chemical Biology and Drug Design

JULY 26, 2023

Abstract Noncoding RNAs (ncRNAs) are engaged in key cell biological and pathological events, and their expression alteration is connected to cancer progression both directly and indirectly. However, there are several limitations, including delivery, uptake, and short half-life, in the application of ncRNAs in cancer treatment.

Engineering 100

Engineering Therapies Cell Biology Drugs 100

FDA Law Blog: Drug Discovery

DECEMBER 7, 2022

Valentine — On November 22, 2022, FDA approved CSL Behring’s BLA for Hemgenix (etranacogene dezaparvovec), an AAV-based gene therapy for the treatment of adults with Hemophilia B who currently use Factor IX prophylaxis therapy, have current or historical life-threatening hemorrhage, or have repeated, serious spontaneous bleeding episodes.

Therapies 119

Therapies FDA FDA Approval Treatment 119

Broad Institute

DECEMBER 18, 2023

But a new path for bringing treatments to patients is starting to emerge. One of our long abiding concerns has been the pharmaceutical industry's progressive exit from R&D on treatments for central nervous system disorders apart from Alzheimer's and for large companies a nearly complete exit from new research in psychiatry.

Drug Development 130

Drug Development Clinical Trials Drugs Disease 130

ASPET

NOVEMBER 30, 2023

In this study, we evaluated the long-term efficacy of novel synthetic neurosteroids in preventing the development of chronic epilepsy and hyperexcitable ictal events in a rat OP model of SE. Similarly, the neurosteroid analog VX significantly attenuated SRS, discharges, HFOs, and ictal events.

Disease 100

Disease Therapies Treatment 100

The Pharma Data

MARCH 29, 2022

Application based on CheckMate -816, the only Phase 3 trial to demonstrate improved event-free survival and pathologic complete response with an immunotherapy-based combination in the neoadjuvant setting of NSCLC. Opdivo -based treatments have shown clinical benefit in four Phase 3 trials in earlier stages of cancer.

Treatment 52

Treatment Clinical Trials Trials Immune Response 52

DrugBank

SEPTEMBER 19, 2024

It offers convenience for patients, promotes medication adherence, and improves treatment outcomes. Achieving a sustained and targeted drug release profile is essential for maximizing therapeutic effects while minimizing the risk of adverse events. This could aid in treatment for diabetic patients who currently rely on injections.

Drugs 98

Drugs Pharmacokinetics Treatment Disease 98

The Pharma Data

APRIL 26, 2022

This approval follows the recent sNDA approval for Veklury for the treatment of non-hospitalized adult and adolescent patients who are at high risk of progression to severe COVID-19. For hospitalized pediatric patients who do not require invasive mechanical ventilation and/or ECMO, a 5-day treatment course is recommended.

Treatment 52

Treatment Hospitals Science Licensing 52

National Drug & Alcohol Research Centre Blog

FEBRUARY 12, 2024

Are these inexperienced drinkers with a low tolerance or seasoned users with long histories of alcohol problems, perhaps receiving treatment for alcohol dependence? What types of alcohol were being consumed before the fatal event?” Only seven people were in a treatment programme at the time they died.

Disease 110

Disease Treatment Drugs Research 110

On Medicine

SEPTEMBER 12, 2023

Considering such findings, the United Nations Office on Drugs and Crime (UNODC) combined the TRT intervention with parenting strategies designed to enhance the carer’s capacity to support children through life events such as disasters, conflicts, and displacement. TRT + Parenting was trialled in Lebanon with Syrian families.

Trials 107

Trials Treatment Research Clinical Trials 107