Drug Target Review

FEBRUARY 14, 2024

Researchers from the Michigan State University have created an advanced human heart organoid system that models the characteristics of pregestational diabetes-induced congenital heart disease found in mice and humans. The most common type of congenital defect in humans is congenital heart disease.

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Disease Research Drugs 59

Drug Target Review

MAY 7, 2024

Why are biomarkers particularly critical in the context of rare diseases? Developing novel therapeutic approaches for rare diseases poses inherent challenges due to the limited populations available for testing. How do AI-enhanced approaches accelerate the drug development process for rare diseases?

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Disease Clinical Trials Clinical Pharmacology Trials 59

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.

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Disease Clinical Trials Therapies Science 59

Drugs.com

SEPTEMBER 6, 2023

Between 1999 and 2020, deaths from heart disease linked to obesity tripled in the United States, and some groups. WEDNESDAY, Sept. 6, 2023 -- Obesity taxes many parts of the body, but new research suggests the heart might take the hardest hit of all.

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Disease Research 105

Drug Discovery Today

MAY 21, 2020

Glasgow, Scotland, 21 May 2020 - Amphista Therapeutics, a biopharmaceutical company creating first-in-class cancer therapeutics that harness the body’s natural processes to remove disease causing proteins selectively and efficiently, today announced the appointment of Dr Ian Churcher as Chief Scientific Officer (CSO).

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Disease 100

FDA Law Blog: Drug Discovery

DECEMBER 13, 2023

Sasinowski — On December 12, 2023, FDA announced the creation of a new advisory committee specifically for treatments for genetic metabolic diseases, the Genetic Metabolic Diseases Advisory Committee, or “GeMDAC.” There are hundreds of known genetic metabolic diseases, most of which are rare and carry significant morbidity.

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Disease Production FDA Drug Development 59

Drug Discovery Today

JULY 15, 2020

Amsterdam, The Netherlands, 23 June 2020 - Scenic Biotech BV (“Scenic”), a pioneer in the discovery of genetic modifiers to enable the development of disease modifying therapeutics for rare genetic disorders and other devastating illnesses, today announced that it has been awarded a €3.1

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Government Disease 100

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. 2020 Jan 1;57(1):372–92. References: Ninou I, Magkrioti C, Aidinis V.

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Clinical Trials Trials Small Molecule Disease 134

Drug Target Review

FEBRUARY 2, 2024

What specific limitations in mirroring diseases in humans are commonly observed when using animal models for preclinical research? Our cells are fully differentiated, non-tumour, primary cells, which have a more natural growth pattern and gene expression profile, and the disease signature is often carried over epigenetically.

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Drugs Disease Clinical Trials Science 116

Broad Institute

APRIL 25, 2024

He started to imagine how the deadly and contagious disease, if confirmed, might spread to half the city’s population. Eight of the 20 patients died, but the spread of the disease in Nigeria stopped there. Blood and urine samples from the man were waiting for Happi in his lab. Happi felt chilled. They called the idea Sentinel.

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Virus Hospitals Disease Government 132

Drug Discovery Today

APRIL 25, 2020

AUSTIN, Texas, April 23, 2020 /PRNewswire/ -- VolitionRx Limited (NYSE AMERICAN: VNRX) ("Volition") today announced that it is actively developing a COVID-19 triage test aiming to predict the likelihood that an individual who is COVID-19 positive will develop complications and severe disease, using its propriety Nu.QTM platform.

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Hospitals Disease 100

The Pharma Data

NOVEMBER 9, 2020

DAEGU, KOREA, November 09, 2020 /24-7PressRelease/ — South Korean religious group Shincheonji Church of Jesus has announced their third convalescent plasma donation drive to take place for 3 weeks starting November 16th at an Indoor Athletics Center in Daegu. Approximately 4,000 members of the group are expected to participate.

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Treatment Vaccine Disease 145

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.

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Disease Clinical Trials FDA Approval FDA 52

The Premier Consulting Blog

JUNE 18, 2023

One exciting application of these technologies is the use of in silico trials in the development of novel therapies for rare diseases. However, with rare diseases there may be no available treatments that could serve as SoC or active control in a clinical trial and assigning patients to placebo may be unethical.

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Drug Development Disease Clinical Trials Drugs 52

FDA Law Blog: Biosimilars

OCTOBER 13, 2024

Tobolowsky — The Rare Pediatric Disease Priority Review Voucher program has had a bit of a tumultuous history in its 12 short years of existence. Over 50 rare pediatric disease priority review vouchers have been awarded to date. Notably, it was reauthorized in 2016 (until 2020) and in 2020 (until 2024).

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Disease FDA FDA Approval Government 64

Drug Target Review

JANUARY 15, 2024

Chronic obstructive pulmonary disease Chronic obstructive pulmonary disease (COPD) is the third leading cause of global deaths. The Pathology of Chronic Obstructive Pulmonary Disease. Annual Review of Pathology: Mechanisms of Disease. 2020 October 1 [2024 January 5]; 27(4):523-31. References 1 Hogg JC, Timens W.

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Immune Response Disease Science Therapies 113

Drug Target Review

SEPTEMBER 21, 2023

Some report that they believe that if they don’t take the medication, they may develop onchocerciasis-related severe skin diseases and may go blind (river blindness) and would prefer a treatment that does not repeat every year. 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

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

SCIENMAG: Medicine & Health

JUNE 23, 2023

are 40% more likely to develop heart disease and have a 30% higher risk of stroke than people who live in urban areas, according to data reported in the 2020 Call to Action: Rural Health: A Presidential Advisory From the American Heart […] DALLAS, June 23, 2023 — People who live in rural areas of the U.S.

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Research Disease 81

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.

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RNA Regulations Disease DNA 98

The Pharma Data

MAY 31, 2022

BIIB122 is an inhibitor of LRRK2, a potential novel target intended to impact the underlying biology and slow the progression of Parkinson’s disease Phase 2b LUMA to enroll approximately 640 participants with early-stage Parkinson’s disease; most advanced clinical study of a LRRK2 inhibitor. Denali Therapeutics Inc.

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Disease Small Molecule Engineering Biosimilars 52

addgene Blog

FEBRUARY 13, 2024

Monoclonal antibody drugs are popular therapeutics for a plethora of disease conditions, from cancer to autoimmune disorders. Antibodies administered as drugs are still immunogenic, meaning that they elicit an immune response from the body.

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Immune Response Drugs Production Disease 75

Drug Target Review

JULY 6, 2023

million scientific procedures involving live animals were carried out in 2020. Three-dimensional organoids have been a popular choice in testing drugs to treat cancer and disease. Animal testing plays a significant role in pre-clinical research and therefore requires the use of millions of animals. In Britain, 2.88

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Animal Testing Drugs Clinical Trials Clinical Research 105

Drug Target Review

OCTOBER 5, 2023

The research team identified 231 individuals diagnosed with relapsing-remitting MS, with 174 of them having undergone aHSCT treatment before the year 2020. Around three years following their aHSCT, 20 patients (11 percent) were administered disease-modifying drugs.

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Therapies Treatment Clinical Trials Disease 116

BioPharma Drive: Drug Pricing

AUGUST 3, 2023

The biotech is acquiring rights to an RNA interference medicine Alnylam invented for a rare blood disorder, continuing a corporate makeover that began in 2020.

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RNA Disease 52

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.

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Hospitals Virus Medical Schools Science 137

The Pharma Data

MARCH 16, 2022

(Nasdaq: BIIB) announced that The Journal of Prevention of Alzheimer’s Disease (JPAD) today published a peer-reviewed manuscript detailing data from the pivotal Phase 3 EMERGE and ENGAGE trials for ADUHELM ® (aducanumab-avwa) 100 mg/mL injection for intravenous use in early Alzheimer’s disease.

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Disease Clinical Trials Treatment Trials 52

New Drug Approvals

SEPTEMBER 29, 2024

gingivalis has been called a risk factor for Alzheimer’s disease ( Kanagasingam et al., gingivalis is associated with cardiovascular disease. Both were reversed by treatment with COR388 ( 2020 AAIC abstract ). A dental substudy of 228 participants is assessing effects of COR388 on periodontal disease. and Europe.

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Small Molecule DNA Disease Trials 62

Drug Target Review

MARCH 19, 2024

While there has been some epidemiological support for vitamin A or deficiency in observational studies, we supported this role for the vitamin in schizophrenia using genetic approaches back in 2020. In other words, a disease state may cause a change in vitamin A levels rather than the vitamin A levels causing a change in disease state.

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Laboratories Bioinformatics International Research Laboratories 105

New Drug Approvals

SEPTEMBER 23, 2024

Target: DNA/RNA Synthesis Pathway: Cell Cycle/DNA Damage Huntington’s disease (HD) is a progressive, autosomal dominant neurodegenerative disorder of the brain, having symptoms characterized by involuntary movements, cognitive impairment, and mental deterioration. 2020-01-02. SCHEME PATENT PTC Therapeutics Inc., Audiol Res. J Pers Med.

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Small Molecule RNA DNA Disease 57

National Institute on Drug Abuse: Nora's Blog

SEPTEMBER 25, 2024

3 The CDC estimates that more than 178,000 people die in the United States each year from diseases attributable to excessive alcohol use, 4 and more than 480,000 people die annually from causes attributable to tobacco use. link] Centers for Disease Control and Prevention. 2020; 66(2):247-254. Published online February 24, 2020.

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Drugs Production Science Marketing 112

Drug Target Review

AUGUST 11, 2023

By selecting specific segments of proteins that elicit robust immune reactions, these vaccines could offer enhanced protection against diseases. Elsevier; 2020 [cited 2023 Jul 25]. 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

SCIENMAG: Medicine & Health

JULY 5, 2023

New drugs are often used not only for one disease (first approved indication) but also for other diseases (supplemental indications).

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Treatment Drugs Disease 67

The Pharma Data

APRIL 7, 2022

These coverage restrictions, including the distinction between accelerated approval and traditional approval, have never been applied to FDA-approved medicines for other disease areas. ADUHELM is indicated for the treatment of Alzheimer’s disease. It may also limit coverage for any future approved treatment in the class. About Biogen.

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Treatment Therapies Disease FDA Approval 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

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 97

Drug Target Review

JUNE 8, 2023

An important research area at AbbVie involves blood cancers, where our ultimate goal is to transform standards of care across these diseases and advance a dynamic cancer research and treatment pipeline. Chronic lymphocytic leukemia in 2020: a surfeit of riches? 2020 Aug;34(8):1979-1983. Epub 2020 May 11.

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Treatment Organic Chemistry Research Disease 105