Drug Target Review

MAY 18, 2023

In sickle cell disease, however, this function is impaired, leading to a range of potential health consequences. The irregularly shaped and sticky red blood cells produced by the disease can obstruct small blood vessels, causing recurring painful attacks. Hydroxyurea, the first sickle cell drug approved by the U.S.

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The Pharma Data

MAY 27, 2022

billion in Medicare funds in 2019 on drugs whose clinical benefits have yet to be confirmed by the Food and Drug Administration, a new study led by researchers from the Johns Hopkins Bloomberg School of Public Health suggests. billion on 36 of these drugs across 55 indications in 2019. federal government spent an estimated $1.8

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Clinical Trials FDA Drugs Government 52

Drug Discovery Today

FEBRUARY 12, 2020

Newly developed recombinant antigens derived specifically from 2019-nCoV for use in infectious disease research, and assay and vaccine development

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Vaccine Disease Research 100

SCIENMAG: Medicine & Health

JUNE 8, 2023

Statement Highlights: A new American Heart Association scientific statement focuses on treatment strategies for pediatric cardiomyopathy (diseases of the heart muscle’s structure and function that may lead to heart failure and death) and is a companion to a 2019 scientific statement focused on diagnosis of the condition.

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Treatment Disease 61

BioPharma Drive: Drug Pricing

AUGUST 28, 2023

The treatment, developed by a biotech Bayer acquired in 2019, appeared safe in a small trial and showed signs of an effect on disease progression.

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Therapies Trials Treatment Disease 98

SCIENMAG: Medicine & Health

AUGUST 15, 2023

. — Exposure to environmental chemicals, including those in common plastic products, has been linked with an increased risk of cardiovascular disease, or CVD, the leading cause of death worldwide. million people died from CVDs in 2019. According to the World Health Organization, 17.9

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Disease Science Production 60

Elrig

APRIL 11, 2024

One of the great highlights of a:head’s work has been the ability to observe disease-specific phenotypes and profile clinically used therapeutics for their ability to alter neuronal network activity. The post How to develop drugs for neurological diseases | Insights from Josh Bagley first appeared on ELRIG.

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Disease Drugs Regulations Therapies 59

Broad Institute

NOVEMBER 4, 2024

By Lucy Mutz November 4, 2024 Credit: Juliana Sohn Kasper Lage is managing director of the Novo Nordisk Foundation Center for Genomic Mechanisms of Disease. The overall goal is to translate genetic findings into biological mechanisms underlying disease that could be targeted with new medicines.

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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. We believe we are at a breakthrough moment in Alzheimer’s disease research.

Disease 59

Disease Clinical Trials Therapies Science 59

Broad Institute

JANUARY 8, 2024

The team used their new system to correct disease-causing mutations in the eyes of two mouse models of genetic blindness, partially restoring their vision. Delivery dilemma Gene editing approaches promise to treat a range of diseases by precisely correcting genetic mutations that cause disease. By Sarah C.P.

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Engineering Research Virus DNA 144

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. They could be spread by physical contact, airborne, waterborne, foodborne, or vector-borne.

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Drugs RNA DNA Disease 111

Advarra

AUGUST 16, 2022

A rare, or orphan, disease by definition affects a small percentage of the population — fewer than 200,000 people in the U.S. But the numbers add up, and taken together, rare diseases impact an estimated 30 million Americans. Since then, the FDA has significantly changed its approach to rare and orphan diseases.

Disease 52

Disease Clinical Trials FDA Approval FDA 52

Broad Institute

JULY 10, 2023

Credit: Allison Colorado, Broad Communications Arriving in the Bay Area after a childhood in increasingly diverse surroundings, Martin became acutely aware of health disparities and the profound differences in disease prevalence among different populations. I wanted to know how I could have any impact on studying genetic diversity.”

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Bioinformatics Disease Medical Schools Research 98

Chemical Biology and Drug Design

NOVEMBER 13, 2023

SARS-CoV-2 is responsible for the coronavirus disease of 2019 (COVID-19) which is one of the most widespread and powerful infections affecting human lungs. WHO proclaimed the outbreak of the Ebola virus disease (EVD), in 2014 that killed hundreds of people in West Africa.

Vaccine 100

Vaccine Laboratories RNA Clinical Development 100

Broad Institute

AUGUST 31, 2023

Related News Next generation prime editing systems move closer to possible therapeutic applications New CRISPR genome editing system offers a wide range of versatility in human cells Prime editing technologies allow scientists to precisely edit the genome in a variety of ways and could one day be used to treat genetic diseases.

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Drug Target Review

NOVEMBER 1, 2023

2 Implication of ATX in a large range of human diseases have been highlighted by both fundamental research and clinical trials. 1-5 Implication of ATX in a large range of human diseases have been highlighted by both fundamental research and clinical trials. Andrew McCarthy Andrew is a Team Leader at EMBL Grenoble since 2007.

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Clinical Trials Small Molecule Trials Disease 134

Broad Institute

JANUARY 30, 2024

Bhattacharyya wears many coats today, splitting his time between the Broad and the infectious disease ward at MGH, where a diagnostic method he developed at Broad is now being tested to see if it helps patients quickly receive the best antibiotics for their infections. million deaths attributed directly to resistant infections.

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Hospitals Medical Schools Virus Science 137

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

Chemical Biology and Drug Design

OCTOBER 5, 2023

Abstract Sickle cell disease (SCD) is the most common genetic disorder, affecting millions of people worldwide. However, the aldehyde functional group metabolic instability has severly hampered their development, except for voxelotor, which was approved in 2019 for SCD treatment.

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Metabolic Stability Treatment Disease Research 100

Drug Target Review

AUGUST 9, 2023

Published on bit.bio’s website, this article authored by Dr Tony Oosterveen dives into the close relationship between FTD and ALS with a focus on mechanisms underlying the neuropathology of both diseases. ALS is the most common motor neuron disease and is estimated to affect more than 80,000 people worldwide.

RNA 98

RNA Regulations Disease DNA 98

Drug Target Review

DECEMBER 9, 2024

Its applications include studying disease pathology to develop targeted therapeutics, visualising enzymes that can decompose plastics, and engineering solutions to antibiotic resistance, among many others. David Baker is listed as an inventor on many patents, focused on compositions and methods for treating particular diseases.

Engineering 105

Engineering International Drug Development Science 105

Advarra

AUGUST 10, 2023

Rapid growth in gene therapy is expected to receive additional support as the Food and Drug Administration (FDA) Center for Biologics Evaluation and Research (CBER) prepares to launch Operation Warp Speed for Rare Diseases. Now, FDA’s CBER is setting its sights on making ambitious strides toward tackling rare diseases.

Drug Development 52

Drug Development Disease Therapies Engineering 52

PLOS: DNA Science

APRIL 18, 2024

Clues to combatting a devastating disease can come from identifying people who have gene variants – mutations – that protect them, by slowing the illness or lowering the risk that it develops in the first place. Gene #1: The Famous Case of Aliria from the Colombian Family In 2019. Quiroz, Ph.D., Quiroz, Ph.D.,

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Disease Research Therapies DNA 98

Drug Target Review

AUGUST 11, 2023

Since December 2019, SARS-CoV-2 (COVID-19) infection has become a worldwide urgent public health concern. Since December 2019, SARS-CoV-2 (COVID-19) infection has become a worldwide urgent public health concern. References Mehdi Hassanpour, et al.

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Vaccine Immune Response Bioinformatics Virus 98

PLOS: DNA Science

MAY 16, 2024

These factors are converging to enable both identification of novel infectious diseases as well as microbial resistance, before these threats can impact public health, write a team from the European Society for Clinical Microbiology and Infectious Diseases in Frontiers in Science. COVID clearly caught us off guard.

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Virus DNA RNA Laboratories 98

LifeSciVC

NOVEMBER 17, 2023

This year we start with a deep dive into the biomedical innovation across our industry – “putting the Big back into Big Pharma” with obesity and Alzheimer’s, and the state of play across other disease areas, industry R&D productivity, and the risks facing the sector. As usual, we close with a brief update on Atlas itself.

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Science Marketing Production Disease 96

The Pharma Data

MAY 31, 2022

the completion of enrollment of its Phase 1 (Ph1), open-label trial of pluripotent stem cell-derived dopaminergic neurons in patients with Parkinson’s disease (PD). of BRT-DA01 in patients with Parkinson’s disease.?. The company was fully acquired by Bayer in 2019. The purpose of the?Ph1 preliminary?efficacy?of

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Trials Disease Clinical Trials Engineering 52

The Pharma Data

APRIL 7, 2021

Sutimlimab, a first-in-class investigational C1s inhibitor, has the potential to be the first approved treatment for hemolysis in adults with cold agglutinin disease, a serious and chronic autoimmune hemolytic anemia. Sutimlimab has the potential to address a major unmet medical need for people with cold agglutinin disease.”.

Disease 52

Disease Trials Treatment Therapies 52

Broad Institute

SEPTEMBER 15, 2023

We apply STACI to analyze the spatio-temporal progression of Alzheimer’s disease and identify the associated nuclear morphometric and coupled gene expression features.

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Disease 40

Covalent Modifiers

JANUARY 14, 2024

Piedra, and Yongcheng Song ACS Infectious Diseases 2023 DOI: 10.1021/acsinfecdis.3c00565 3c00565 Highly contagious SARS-CoV-2 coronavirus has infected billions of people worldwide with flu-like symptoms since its emergence in 2019. It has caused deaths of several million people.

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Virus Drug Development Disease Drugs 75

PLOS: DNA Science

OCTOBER 19, 2023

Also recently, FDA’s Cellular, Tissue, and Gene Therapies Advisory Committe turned down a stem cell treatment for amyotrophic lateral sclerosis, aka ALS, Lou Gehrig’s disease, or motor neuron disease. ALS is a disease of adulthood, beginning typically between 40 and 70 years of age. Most ALS cases are sporadic.

Therapies 111

Therapies FDA Clinical Trials DNA 111

Broad Institute

JUNE 21, 2023

With further development, the findings could inform a potential treatment that lowers oxygen levels in certain tissues in humans to reverse advanced disease. If we accept that disease stems from the interaction of genes and environment, then why not manipulate the environment?” The study appeared recently in Human Molecular Genetics.

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Treatment Disease Molecular Biology Medical Schools 98

Drug Target Review

APRIL 26, 2023

Gene-editing methods allow researchers to modify DNA, resulting in alterations in physical characteristics such as eye colour and susceptibility to diseases. 1 RNA-delivery particles have the potential to revolutionise the treatment of lung diseases such as cystic fibrosis.

RNA 98

RNA Medical Schools Engineering DNA 98

Drug Target Review

OCTOBER 12, 2023

Published in Nature Genetics , scientists from the University of North Carolina at Chapel Hill and the University of California, San Francisco discovered the connections of risk variants with functions in microglia and how they might contribute to Alzheimer’s disease (AD). 2019 August 30 [cited 2023 October 6]; 11(233):20.

Regulations 103

Regulations DNA Disease Research 103

Conversations in Drug Development Trends

FEBRUARY 22, 2023

Rare Disease Day on February 28th is a chance for rare disease communities to come together as one and join in conversation with stakeholders searching for treatments, raising money to support research, and changing the way healthcare and governments interact with patients. In my case, my daughter Iris has GM1 gangliosidosis.

Disease 52

Disease Clinical Trials Trials Treatment 52

Drug Target Review

SEPTEMBER 26, 2023

Human derived induced pluripotent stem cells (hiPSCs) have revolutionised research and are increasingly used for toxicology screening and disease modelling. 2019 Mar 1;55:93–100. 2019 Nov 1;8(6):784–8. Using stem cell-derived neurons in drug screening for neurological diseases. 2019 Jun;78:130–41. Toxicol In Vitro.

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Drugs FDA Disease Animal Testing 111

New Drug Approvals

SEPTEMBER 29, 2024

gingivalis has been called a risk factor for Alzheimer’s disease ( Kanagasingam et al., gingivalis DNA was detected in postmortem cortices from people with AD and healthy controls, and in CSF of AD patients ( Jan 2019 news on Dominy et al., gingivalis is associated with cardiovascular disease.

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Small Molecule DNA Disease Trials 62