Drug Target Review

DECEMBER 29, 2023

Scientists in China have published their findings 1 regarding novel biomarkers, which they hope will benefit patients by identifying the disease at an earlier stage. Diabetic kidney disease DKD is a major microvascular complication of type 2 diabetes mellitus (T2DM). References Du S, Zhai L, Ye S, et al. Science China Life Sciences.

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Antidote

FEBRUARY 8, 2023

Chronic obstructive pulmonary disease, known commonly as COPD, is a condition that impacts more than 16 million Americans.

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Drug Target Review

MARCH 12, 2024

The study’s findings offer new insights into the biochemical language microbes use to influence distant organ systems, which could revolutionise the way researchers approach disease. microbeMAAST: a taxonomically informed mass spectrometry search tool for microbial metabolomics data. This study was published in Cell Reports.

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Conversations in Drug Development Trends

MAY 14, 2024

By: Amy Raymond, Derek Ansel, Nathan Chadwick, & Juliane Mills When choosing a CRO for a rare disease study, what truly sets them apart is their methodology: the CRO’s mindset, their approach to each unique study, and their agility in navigating the inherent complexities of rare disease research. Common goals empower change.

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Conversations in Drug Development Trends

AUGUST 29, 2024

By: Nathan Chadwick, Therapeutic Strategy Lead, Rare Disease Over the last few years in clinical trials, particularly within the rare disease community, a notable shift is underway, where patients and caregivers are taking the lead in reaching out to clinical trial sites rather than the other way around.

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Advarra

SEPTEMBER 29, 2022

Informed consent is one of the central protections the regulations provide to research subjects. This tip sheet outlines the regulatory requirements for research informed consent forms (ICFs). The regulatory requirements for informed consent will vary depending upon which regulations apply to the conduct of a particular study.

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Broad Institute

JANUARY 25, 2024

They found significant associations between the thinning of different retinal layers and increased risk of developing ocular, cardiac, pulmonary, metabolic, and neuropsychiatric diseases and identified genes that are associated with retinal layer thickness. Their findings are published in Science Translational Medicine. “We

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Antidote

JANUARY 12, 2023

Alzheimer’s disease is the most common type of dementia in the United States, impacting around 6.2 Since it was first observed in the early 1900s, a lot of information has been uncovered through medical research. million people above the age of 65.

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DrugBank

JULY 20, 2023

Rare diseases, therefore, present compelling opportunities for Drug Development. Since each disease is present in small groups of patients, this research is also fraught with complex challenges. Limited numbers of patients translate to scarce information, making this research risky and expensive. –5.9%

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Conversations in Drug Development Trends

JANUARY 24, 2024

Written By: Derek Ansel, MS, CCRA, Executive Director, Therapeutic Strategy Lead, Rare Disease Given that 80% of rare diseases have a genetic etiology, genetic implications should be addressed at the onset of a clinical program to support trial enrollment.

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PPD

OCTOBER 12, 2023

Since chimeric antigen receptor T cell (CAR-T) therapy was first approved in 2017, there has been a marked increase of cell and gene therapy studies resulting in significant changes in the way diseases are treated as well as patient outcomes. The post Informed Design of Bioanalytical PCR Assay Testing Parameters appeared first on PPD Inc.

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Fierce BioTech

JULY 25, 2023

How combining datasets from multiple sources can inform and improve commercial strategies Hundreds of rare disease treatments have entered the market over the past decade—thanks largely to a combination of government incentives, strong urging from patient advocacy groups and advances in cell and gene therapies.

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Agency IQ

JUNE 26, 2023

FDA quietly kicks off process that could inform future rare disease regulatory reforms Reforms buried within the FY2023 omnibus spending bill require the development of a new report looking at the regulatory processes of the U.S. Meanwhile, in the EU rare diseases are defined using a different set of criteria. In the U.S.,

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Agency IQ

JUNE 21, 2024

BY ALEXANDER GAFFNEY, MS, RAC This week, PETER MARKS, director of FDA’s Center for Biologics Evaluation and Research (CBER), spoke at several sessions of the Drug Information Association (DIA) annual meeting. AgencyIQ has the highlights, with new information on a “Rare Disease Hub” modeled as a sort of Center of Excellence for rare diseases.

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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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Fierce BioTech

APRIL 17, 2024

RWD’s Value in Accelerating Autoimmune Disease Diagnosis swheeler Wed, 04/17/2024 - 17:17 Wed, 06/05/2024 - 14:00 Resource Type Webinar Duration 60 minutes Delayed diagnosis of autoimmune diseases is common due to subjective symptoms, lack of definitive testing, and gender bias. Click here to login.

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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 treatment often initially provides good symptom relief and those affected by the disease can live a largely normal life.

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Conversations in Drug Development Trends

MAY 14, 2024

By: Amy Raymond, Derek Ansel, Nathan Chadwick, & Juliane Mills When choosing a CRO for a rare disease study, what truly sets them apart is their methodology: the CRO’s mindset, their approach to each unique study, and their agility in navigating the inherent complexities of rare disease research. Common goals empower change.

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Broad Institute

OCTOBER 23, 2023

By Makenzie Kohler October 23, 2023 People of South Asian ancestry around the world have more than double the risk of developing cardiometabolic diseases like diabetes, heart attack, and stroke compared to other populations. They will also have the opportunity to inform the researchers on whether their health status changes over time.

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Broad Institute

AUGUST 17, 2023

Chao joined the NIH as a Post-Baccalaureate Intramural Research Training Award fellow, where she processed and analyzed genomic data in the context of rare disease. In that role, Chao continued to work in rare disease genomic analysis, identifying sections of duplicated or deleted genomic sequences called copy number variants.

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SCIENMAG: Medicine & Health

AUGUST 7, 2023

Philadelphia, August 8, 2023 – There is a need to better deliver information on medical nutrition therapy for patients with Parkinson’s disease (PD).

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Drug Target Review

APRIL 4, 2024

What are the key findings of the preclinical study regarding the neurophysiological brain state in a Parkinson’s disease psychosis (PDP) model? The animal model is based on creating a dopamine-deficient state, by a specific lesion of dopaminergic cells, and then exposing it to repeated substitution therapy with L-DOPA.

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The ChEMBL-og

FEBRUARY 22, 2021

Updated drug safety information is available (as of ChEMBL 28 ) for drugs with boxed warnings and for withdrawn drugs. Therefore, toxicity class(es) have been assigned to approved drugs with boxed warning information described on medicinal product labels (e.g. The drug safety information will be updated in future database releases.

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The Pharma Data

MAY 14, 2021

Product Name: Home – Beat Kidney Disease – 2020 – Beat Kidney Disease. Home – Beat Kidney Disease – 2020 – Beat Kidney Disease is backed with a 60 Day No Questions Asked Money Back Guarantee. This product is not intended to diagnose, treat, cure, or prevent any disease.

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SCIENMAG: Medicine & Health

AUGUST 23, 2023

Research published in the Journal of the American Heart Association reveals that many US young adults have poor lifestyle factors and cardiometabolic diseases—such as obesity, diabetes, and hypertension—with varying rates based on race and ethnicity.

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FDA Law Blog: Drug Discovery

MAY 19, 2024

By Sarah Wicks — On May 21, 2024, the EveryLife Foundation for Rare Diseases (ELF) will host a Scientific Workshop at the National Press Club in Washington, D.C. aimed at identifying and characterizing the challenges in developing therapies for ultra-rare diseases and conditions that affect exceedingly small populations.

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Chemical Biology and Drug Design

JANUARY 15, 2024

The expression levels of endoplasmic reticulum (ER) stress-related protein and silent information regulator 1/nuclear factor kappa-B (Sirt1/NF-κB) signaling pathway were used to observe the therapeutic effect of naringin.

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SCIENMAG: Medicine & Health

JUNE 16, 2023

Breast cancer is a complex disease, and its progression is difficult, yet important, to predict. Credit: UMass Amherst Breast cancer is a complex disease, and its progression is difficult, yet important, to predict.

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PLOS: DNA Science

JULY 27, 2023

That information led, thanks to vaccine shelved from the first SARS circa 2003, to the rapid development and deployment of mRNA vaccines against the new infectious disease. which spit out information guiding the trimming of tumors in real time, a bit reminiscent of its ancestor Hal the computer from 2001: A Space Odyssey.

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The Pharma Data

JULY 8, 2021

ADUHELM should be initiated in patients with mild cognitive impairment due to Alzheimer’s disease or mild Alzheimer’s dementia. The update includes an addition to the Indications and Usage section of the label (Section 1) to emphasize the disease stages studied in the clinical trials, as seen below ( italics to note updated language).

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The Pharma Data

MAY 22, 2023

WHO launches global network to detect and prevent infectious disease threats WHO and partners are launching a global network to help protect people from infectious disease threats through the power of pathogen genomics. Diseases do not respect borders: a disease threat in one country is also a threat to others.

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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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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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The Pharma Data

JUNE 25, 2021

We are committed to responding to questions from the Alzheimer’s disease community and providing more details about our plans. ADUHELM is indicated for the treatment of Alzheimer’s disease. Please see Important Safety Information below. For more information, please contact Biogen Support Services at 1-833-425-9360.

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The Premier Consulting Blog

OCTOBER 11, 2022

Clinical trials for ultra-rare diseases can be particularly challenging to mount due to small, geographically-dispersed patient populations. In this article, we review two case studies involving the successful use of RWD or RWE in advancing the clinical development of treatments for rare diseases. Reference: [1] Hansen, et al.

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The Pharma Data

OCTOBER 27, 2020

Infectious Disease Least Severe in School-Aged Children. 27, 2020 — School-aged children seem to have the least severe disease for different infectious diseases, according to a study published online Oct. Professional. TUESDAY, Oct. 15 in Scientific Data. Glynn, Ph.D., ” Abstract/Full Text. © 2020 HealthDay.

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On Medicine

FEBRUARY 26, 2023

Behçet’s disease is a rare multisystem vasculitic disorder that can affect any of the blood vessels of the body; signs and symptoms are diverse among individuals and range in severity, but often present as recurrent oral aphthous ulcers, genital ulcers, or as complications involving the eyes.

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