Broad Institute

FEBRUARY 7, 2024

However, researchers lack efficient ways to explore how these genetic variants are molecularly connected to cardiovascular disease, limiting efforts to develop therapeutics. The team found that a key biological mechanism involved in a rare vascular disease may influence CAD risk.

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The Pharma Data

JULY 23, 2021

Nasdaq: BIIB) today announced it will share multiple oral and poster presentations from its Alzheimer’s disease clinical development portfolio at the Alzheimer’s Association International Conference (AAIC), which will be held in Denver, Colorado and online July 26-30, 2021. ADUHELM is indicated for the treatment of Alzheimer’s disease.

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International Disease Clinical Trials Trials 52

Chemical Biology and Drug Design

JANUARY 15, 2024

Abstract This study was designed to explore the protective effect and mechanism of naringin (NG) on radiation-induced heart disease (RIHD) in rats. The results showed that NG treatment significantly attenuated the 20 Gy XR-induced decline of LVEF and LVFS and the elevation of LVIDs.

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Disease International Regulations Treatment 100

Drug Target Review

JULY 13, 2023

Drug discovery is a complex and vital field that continually seeks to identify new therapeutic targets and develop effective treatments. Dewpoint’s breakthrough was the realisation that certain condensates are associated with human diseases. Klein notes the importance of Dewpoint’s disease-agnostic approach.

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Drug Patent Watch

DECEMBER 9, 2024

High-Throughput Screening: Modern Technology Meets Natural Products Advanced technologies now allow researchers to rapidly test thousands of natural compounds against specific disease targets. Artemisinin: Nobel Prize-Winning Malaria Treatment Artemisinin, derived from the sweet wormwood plant, has become a crucial treatment for malaria.

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Drugs Drug Development Pharmaceuticals Production 97

Codon

NOVEMBER 3, 2024

In the 1924 novel, The Magic Mountain , Thomas Mann describes a sanatorium patient named Anton Ferge as he undergoes a painful tuberculosis (TB) treatment. Physicians working in the early 20th century had little choice but to treat the world’s most rampant infectious disease with methods such as these. million people every year.

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Vaccine Trials Therapies Clinical Trials 92

Drug Target Review

SEPTEMBER 9, 2024

functions to reset the immune system and potentially provide remission for allergic diseases? In allergic diseases, APCs identify and present unknown antigens to the immune system. This can lead to inadequate long-term effectiveness with low rates of disease remission. They also require frequent administration.

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Disease Immune Response Clinical Trials Treatment 64

SCIENMAG: Medicine & Health

OCTOBER 20, 2023

Now an international team led by the Hudson Institute of Medical Research has found a way to determine which species are important and how they interact to create a healthy microbiome. The more diverse species in your gut, the better it is for your health. Credit: Hudson Institute of Medical Research The more diverse species […]

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Treatment Disease International Research 52

Drug Target Review

SEPTEMBER 5, 2024

I was part of building up this small biopharma company and learn a lot about rare diseases, regulatory interactions and challenges related to that, challenges related to out licensing drug candidates and having new people joining the company every month as we were growing so fast.

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Drug Target Review

MARCH 19, 2024

In the current study undertaken by an international team of researchers, co-led by Dr William Reay, we used the genetics of retinol itself as a way to proxy its effect in thousands of traits, including brain structure, vision, and the immune system through causal modelling.

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The Pharma Data

APRIL 12, 2022

WHO welcomes the establishment of a new Heads of State and Government Group to accelerate progress towards the SDG target for noncommunicable diseases ? a one-third reduction in “premature” deaths from diseases like diabetes, cancer, heart and lung disease and the promotion of mental health and well-being.

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International Disease Government Treatment 52

Drug Target Review

OCTOBER 25, 2024

How does precision neuroscience differ from traditional approaches in the treatment of neurodegenerative diseases, and what advantages does it offer? Traditionally, Alzheimer’s disease is diagnosed by clinical examination, medical history and cognitive testing.

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Disease Clinical Trials Therapies Science 59

The Pharma Data

AUGUST 15, 2021

Food and Drug Administration (FDA) has granted Fast Track designation to VTX-801, Vivet’s clinical-stage gene therapy for the treatment of Wilson Disease – a rare, genetic disorder that reduces the ability of the liver and other tissues to regulate copper levels, causing severe hepatic damage, neurological symptoms, and potentially death.

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Disease FDA Treatment Clinical Trials 52

Drug Target Review

DECEMBER 11, 2024

This shift in focus is especially critical in toxicology, where accurate target analysis plays a vital role in identifying toxic effects and ensuring patient safety, particularly as the field transitions from traditional drugs to the promising realm of biotherapeutics, especially for rare diseases.

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International Laboratories Drugs Pharmacokinetics 52

Drug Target Review

DECEMBER 21, 2023

Researchers at the Allen Institute for Cell Science , a division of the Allen Institute in Seattle, US, developed a unique mathematical framework to uncover internal organisation of cellular structures in human induced pluripotent stem cells (hiPSCs) while taking account of the wide range of variation that naturally occurs in cell shapes.

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International Science Disease Research 105

The Pharma Data

JULY 15, 2021

Challenge to Support an Innovative Solution That Addresses Disconnect in Perception of Psoriatic Disease Severity and Undertreatment Documented in Multinational UPLIFT Survey UPLIFT Data Presented at WPPAC 2021 Report Substantial Disease Burden for People Living With Psoriasis and Psoriatic Arthritis.

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International Disease Treatment Science 52

Advarra

MARCH 6, 2025

For more than 35 years, Advarra has been committed to protecting the rights and welfare of clinical trial participants while helping to improve healthcare outcomes, advancing medical knowledge, and bringing innovative, life-extending treatments to market that benefit millions of patients worldwide.

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Clinical Research Research Clinical Trials Bioethics 52

Drug Target Review

NOVEMBER 6, 2023

Advances in research and development and the utilisation of combination treatment strategies are revolutionising the haematology care landscape, but more must be done to address patients’ individual needs. 2 It is this complexity that necessitates powerful, targeted combination therapies.

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Small Molecule Therapies Treatment Disease 111

Drug Target Review

OCTOBER 11, 2023

This momentous discovery has the potential to reshape how we approach the diagnosis, treatment, and prevention of this deadly disease. Prostate cancer is a complex and heterogeneous disease, and the quest to tailor treatment to individual patients has never been more crucial.

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Research Disease Treatment DNA 98

Drug Target Review

DECEMBER 6, 2024

In cases where control or treatment groups show large variability, this could reflect true biological responses to a treatment, and removing such data could reduce the accuracy of the analysis. It is crucial during this phase to strike a balance between removing erroneous data and preserving meaningful biological variability.

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RNA Drugs Research Science 52

PLOS: DNA Science

SEPTEMBER 5, 2024

Diagnosis began to shift from a body-part basis to a molecular one, which I wrote about in “Mutation and location important in cancer treatment” for The Lancet in 2015. The researchers tested the model on 19 types of cancers, and validated the findings among several international patient groups.

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Treatment FDA Approval DNA Small Molecule 71

Drug Target Review

SEPTEMBER 19, 2024

Based on Nobel Prize-winning research, IRLAB has grown rapidly to become recognised and respected as a world-leader in understanding the complex neuropharmacology of CNS disorders, especially Parkinson’s disease (PD). The pipeline currently includes five drug candidates, all of which have the potential to revolutionise the treatment of PD.

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Disease Drugs Treatment Drug Development 52

Conversations in Drug Development Trends

NOVEMBER 3, 2022

But for rare disease trials, which are already challenging to enroll, the new guidance introduces nuances that speak to broader recruitment trends in orphan drugs, suggested Derek Ansel, executive director, therapeutic strategy lead, rare diseases at Worldwide Clinical Trials. medical access, such as social determinants of health.

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Disease Clinical Trials Trials International 52

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

Drug Target Review

SEPTEMBER 21, 2023

The findings supported the possibility put forward by Cotton et al (2016) concerning the chance of repurposing these drugs for adulticidal treatment. The available treatment for that is minor surgery -to remove the nodules. Need for adulticidal drugs More than 99 percent of countries worldwide has been affected by this disease.

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Drugs Treatment Disease Research 105

The Pharma Data

SEPTEMBER 9, 2021

(NYSE and TASE: TEVA), today announced results from a network meta-analysis examining the efficacy of AJOVY (fremanezumab-vfrm), atogepant and Nurtec ® ODT (rimegepant) in the preventive treatment of episodic migraine (EM). 8-12, 2021. Atogepant 60 mg BID. a 1-12 weeks; 9-12 weeks for Rimegepant. b Exact value with 3 decimal places: ?0.004.

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International Treatment Clinical Trials Trials 52

Broad Institute

OCTOBER 23, 2024

They knew that discovering the genetic cause of her disorder would help them find other people like her, help get the condition formally recognized as a new disease, and help them better advocate for research into new treatments. This is the first human disease caused by loss of one copy of a lncRNA gene.

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DNA RNA Disease Hospitals 128

Drug Target Review

JUNE 3, 2024

How does COUR Pharmaceuticals’ immune-modifying nanoparticle platform differ from traditional approaches to treating immune-mediated diseases? Essentially, our CNPs reprogramme the immune system by restoring balance and returning the body to a homeostatic state, offering a precise approach to treating immune-mediated diseases.

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The Pharma Data

AUGUST 30, 2021

Approval marks an important transformation in the treatment of more than 13 million people suffering from chronic kidney disease in Japan. CKD is a serious, progressive condition defined by decreased kidney function and is often associated with an increased risk of heart disease or stroke.

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Treatment Trials Disease Clinical Trials 52

KIF1A

JULY 8, 2023

Each week, Dr. Dylan Verden of KIF1A.ORG summarizes newly published KIF1A-related research and highlights progress in rare disease research and therapeutic development. This year has seen a growing set of successes in the rare disease space, with varying gene editing effects and administration routes that add to a suite of potential tools.

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Therapies Regulations FDA Treatment 98

National Institute on Drug Abuse: Nora's Blog

AUGUST 28, 2024

Over the past two decades, substance use treatment admissions have increased among people aged 50 and over. Chronic pain is prevalent among older adults, and treatment for pain is less effective in this age group than it is in younger people; it negatively impacts quality of life and is a risk factor for cognitive decline and premature death.

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Treatment Research Disease Drugs 110

PPD

AUGUST 5, 2024

Pulmonary arterial hypertension (PAH) is a progressive and fatal lung disease that is caused or influenced by multiple factors. However, it is often the case that the smaller marginal benefit of new treatments leads to a reduction in relevant outcomes that can be used as endpoints. Historically, the available drugs and U.S.

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Clinical Trials Trials Clinical Research Disease 98

Drug Target Review

AUGUST 25, 2023

What makes RRx-001 a first-in-class hypoxia-activated therapeutic, and how does its dual mechanism of action differ from traditional cancer treatments? Most, if not all, traditional cancer treatments are single minded. Several metabolites of RRx-001 (nibrozetone) are under preclinical evaluation.

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Small Molecule Virus International Clinical Trials 98

FDA Law Blog: Biosimilars

DECEMBER 4, 2023

A prescription not issued in the usual course of professional treatment or in legitimate, authorized research is not a prescription within the meaning of the CSA and the pharmacist knowingly filling it, along with the prescriber issuing it, is subject to penalties. 21 U.S.C. § 829; 21 C.F.R. Complaint ¶ 57. (The Complaint ¶ 58.

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ASPET

OCTOBER 20, 2023

The intentional release of chemical warfare agents (CWAs) during World War I caused life-long damage for survivors, and CWA use is outlawed by international treaties. Emergency treatment is limited to supportive care using bronchodilators to control airway constriction, and rescue with mechanical ventilation to improve gas-exchange.

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International Regulations Treatment Disease 100

The Pharma Data

MAY 27, 2022

The FDA’s decision is informed by the results of Pfizer’s nonclinical studies and a Phase 2a clinical study of ervogastat/clesacostat, which showed that treatment with ervogastat/clesacostat reduced liver fat with a favorable safety and tolerability profile. Pfizer Inc. NYSE: PFE) today announced the U.S.

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Treatment FDA Therapies Vaccine 52

Drug Target Review

SEPTEMBER 4, 2023

There have been several studies that have reported the ability of infectious disease agents to be drug resistant. Infectious agent includes bacteria, virus, fungi, and parasites, and they are capable of causing disease in a living organism. International research and symposium help to understand and combat regional challenges.

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