ASPET

SEPTEMBER 29, 2023

Recent studies have yielded controversial results on the long-term effects of statin treatment on the risk of cardiovascular (CV) events. The incidence of first CV events was 6.0% Taken together, our data demonstrate that a 7-year stable control of LDL-C induces a forty percent reduction in the incidence of CV events.

Treatment 100

Treatment Therapies 100

ASPET

SEPTEMBER 20, 2024

Post-traumatic stress disorder (PTSD) is caused by exposure to a traumatic or stressful event. Current pharmacological treatments for PTSD are insufficient, with fewer than 30% of patients reporting symptom remission. Treatments began 4 hours after FC. The OFT was conducted one day before the last FC re-exposure.

Treatment 190

Treatment Research 190

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 69

Clinical Development Clinical Trials Clinical Pharmacology Trials 69

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

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

New Drug Approvals

APRIL 11, 2025

1] [2] Adverse effects The US Food and Drug Administration prescription label for fitusiran contains a boxed warning for thrombotic events (blood clotting) and gallbladder disease (with some recipients requiring gallbladder removal). [2] Fitusiran 1711.0g/mol, 1] It is an antithrombin-directed small interfering ribonucleic acid. [1]

Clinical Trials 107

Clinical Trials FDA FDA Approval Trials 107

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 94

FDA Approval Treatment FDA DNA 94

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

The Pharma Data

JUNE 13, 2025

Currently, treatment options for patients with relapsed or refractory disease remain limited, and prognosis is poor. Standard single-target CD19-directed CAR T-cell therapies typically enable long-term remissions in roughly 40% of patients, emphasizing a significant unmet need for new and more effective treatment strategies.

Therapies 40

Therapies Treatment Clinical Development Trials 40

The Pharma Data

JUNE 18, 2025

The double-blind treatment period lasted 24 weeks, during which both efficacy and adverse events (AEs) were closely monitored. of patients in the atogepant arm discontinued treatment because of AEs, versus a substantially higher 29.6% Specifically, only 12.1% among those taking topiramate. to 0.59; p <0.0001).

Clinical Trials 40

Clinical Trials Treatment Therapies Trials 40

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

The Pharma Data

JUNE 15, 2025

The data, which encompass up to 3 years of follow-up after a single infusion of the treatment, were shared in an oral presentation at the European Academy of Allergy and Clinical Immunology (EAACI) Congress 2025, held from June 13–16 in Glasgow, United Kingdom. President and Chief Executive Officer of Intellia Therapeutics.

Treatment 40

Treatment Therapies Licensing Licensing 40

The Pharma Data

JUNE 12, 2025

The data were revealed during an oral presentation (S137) at the 2025 European Hematology Association (EHA) Congress in Vienna, reflecting ongoing progress in developing new treatment options for patients battling this aggressive hematologic malignancy.

Treatment 40

Treatment Small Molecule Therapies Trials 40

The Pharma Data

JUNE 15, 2025

This is crucial information for guiding both patients and physicians in their treatment decisions.” All patients received their respective biologic therapy in addition to standard background treatment with mometasone furoate nasal spray (MFNS). Serious adverse events were somewhat lower in Dupixent (2%) than in omalizumab (4%).

Treatment 40

Treatment Disease Clinical Trials Trials 40

The Pharma Data

JUNE 16, 2025

Currently, the medial survival for patients with MZL after multiple lines of treatment falls within the range of 3–5 years, reflecting a significant and persistent need for more effective therapeutic options. Neurologic events (NE) of any grade were documented in 33% of patients, with Grade 3 NEs in 4% and no Grade 4 or 5 NEs.

Therapies 40

Therapies Clinical Trials Disease Trials 40

The Pharma Data

JUNE 11, 2025

The investigational therapy, an Fc-free anti-CD40L monoclonal antibody, is being developed for the treatment of systemic lupus erythematosus (SLE), a chronic autoimmune disease with limited treatment options and significant unmet need. Treatment-Emergent Adverse Events (TEAEs) : Occurred in 82.6% in the SOC group.

Disease 40

Disease Treatment Trials Therapies 40

The Pharma Data

JUNE 15, 2025

According to the data, the combination demonstrated a high ORR and a strong depth of response in a patient population with limited treatment options and poor outcomes under standard care. were penta-drug refractory, having been exposed to additional lines of treatment. Among these: 84.4% had previously received a bispecific antibody.

Therapies 40

Therapies Treatment Disease FDA Approval 40

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

The Pharma Data

JUNE 9, 2025

The COACH study is the first clinical investigation to assess the combined effects of two once-weekly treatments—TransCon® CNP (navepegritide) and TransCon® hGH (lonapegsomatropin)—in children with achondroplasia. TransCon CNP as a monotherapy has demonstrated the potential to transform the treatment of achondroplasia,” Dr. Shu stated.

Trials 40

Trials Therapies Treatment FDA 40

FDA Law Blog: Biosimilars

APRIL 13, 2025

LYTGOBI was granted accelerated approval for the treatment of adult patients with previously treated, unresectable, locally advanced, or metastatic intrahepatic cholangiocarcinoma (iCCA) with FGFR2 gene fusions or rearrangements. Join us as we break down what went wrong this time. What Happened? The problem? As a single-arm trial (i.e.,

Drugs 52

Drugs FDA Trials Treatment 52

The Pharma Data

JUNE 12, 2025

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.

Disease 40

Disease Clinical Development Treatment Medical Schools 40

The Pharma Data

JUNE 9, 2025

Food and Drug Administration (FDA) for the treatment of cataplexy or excessive daytime sleepiness (EDS) in patients aged seven years and older with narcolepsy. of participants reported treatment-emergent adverse events (TEAEs), all of which were mild or moderate and consistent with previous safety data on Xywav.

FDA Approval 40

FDA Approval FDA Pharmaceuticals Treatment 40

The Pharma Data

JUNE 11, 2025

Achieving the Primary Endpoint: Sotyktu Demonstrates Superiority Over Placebo The POETYK PsA-1 trial, officially designated as study IM011-054, enrolled patients with active PsA who had not received prior treatment with biologic disease-modifying antirheumatic drugs (bDMARDs).

Trials 40

Trials Treatment Disease Clinical Trials 40

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

The Pharma Data

JUNE 11, 2025

argenx Unveils Positive Phase 2 Results for Efgartigimod in Myositis and Sjogren’s Disease at EULAR 2025 argenx SE a global immunology company dedicated to advancing treatments for severe autoimmune diseases, presented encouraging new clinical data at the 2025 European Congress of Rheumatology (EULAR), held June 11–14 in Barcelona, Spain.

Disease 40

Disease Treatment Trials Therapies 40

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

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

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

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

The Pharma Data

JUNE 6, 2025

This outcome suggests sibeprenlimab’s potential to not only delay disease advancement but also improve long-term renal outcomes, addressing a critical unmet need for patients with IgAN, many of whom currently face limited treatment options. Treatment-emergent adverse events (TEAEs) occurred in 76.3% in the placebo group.

Trials 40

Trials Treatment Therapies FDA 40

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