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.

Treatment 61

Treatment Disease 61

Drug Target Review

JUNE 21, 2023

Most rare diseases are caused by a single gene defect, but severity can vary considerably among patients. Modifier genes can help explain that variability and can alter or even prevent disease onset and progression, making them appealing therapeutic targets. However, the identification of these genes is challenging.

Disease 111

Disease Clinical Trials Therapies Science 111

Drug Target Review

JULY 12, 2023

Molecular-level biochemical assays like transcriptomics, genomics and proteomics have emerged as valuable tools for identifying potential targets in cancer treatment through deep cyclic inhibition (DCI). Importantly, transcriptomics information shows that certain aspects of a disease change the gene expression profile.

Biochemical Assays 98

Biochemical Assays Treatment Bioinformatics Disease 98

Broad Institute

JULY 10, 2024

A three-drug cocktail known as Trikafta has greatly improved patient quality of life since its development in 2019, but can cause cataracts and liver damage and must be taken daily at a cost of about $300,000 per year. With further development, it could pave the way for treatments that are administered only once and have fewer side effects.

Engineering 145

Engineering Packaging DNA Treatment 145

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.

Disease 80

Disease Clinical Trials Drugs Trials 80

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.

Disease 59

Disease Clinical Trials Therapies Science 59

Broad Institute

JUNE 21, 2023

Now, researchers at the Broad Institute of MIT and Harvard have found that treatment with continuous hypoxia — low-oxygen conditions comparable to levels at a Mount Everest base camp — restores balance and coordination in a mouse model of Friedreich’s ataxia. The study appeared recently in Human Molecular Genetics. “If

Treatment 98

Treatment Disease Molecular Biology Medical Schools 98

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.

Metabolic Stability 100

Metabolic Stability Treatment Disease Research 100

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.

Drugs 111

Drugs RNA DNA Disease 111

The Pharma Data

JANUARY 17, 2021

18, 2021 /PRNewswire/ — Aruvant Sciences, a private company focused on developing gene therapies for rare diseases, and Lonza announced today their agreement in support of ARU-1801, Aruvant’s one-time investigational gene therapy for sickle cell disease (SCD). NEW YORK , Jan.

Disease 52

Disease Trials Treatment Clinical Trials 52

The Pharma Data

JANUARY 17, 2021

18, 2021 /PRNewswire/ — Olympus announced that Highmark Blue Cross Blue Shield (BCBS) updated its coverage policy to include the Spiration ® Valve System for eligible patients suffering from severe emphysema, a form of Chronic Obstructive Pulmonary Disease (COPD). . CENTER VALLEY, Pa. , ” . ” .

Treatment 52

Treatment Clinical Trials Trials Disease 52

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.

Drugs 105

Drugs Treatment Disease Research 105

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. The study appears in Nature Biotechnology.

RNA 98

RNA Medical Schools Engineering Disease 98

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.

DNA 128

DNA RNA Disease Hospitals 128

The Pharma Data

FEBRUARY 8, 2021

Janssen Announces Treatment with ERLEADA ® (apalutamide) Significantly Improved Overall Survival in Patients with Metastatic Castration-Sensitive Prostate Cancer. Based on these data, ADT alone should no longer be considered sufficient for patients with advanced, castration-sensitive disease.”.

Treatment 52

Treatment Pharmaceutical Companies Therapies Pharmaceuticals 52

Alta Sciences

APRIL 17, 2024

Five Promising Treatment Areas in Early-Phase Drug Development in 2024 aasimakopoulos Wed, 04/17/2024 - 15:52 Early-phase drug development is an ever-changing landscape, as emerging science leads to new promising areas of research for the treatment of human health issues.

Drug Development 52

Drug Development Treatment FDA Approval Drugs 52

Drug Target Review

AUGUST 29, 2024

However, these treatments will eventually fail for the majority of patients, so there is a strong need for better CAR therapies. How might the CD5 knockout approach impact the treatment of other types of cancers beyond T-cell lymphoma?

Therapies 59

Therapies Treatment Engineering Molecular Biology 59

The Pharma Data

FEBRUARY 22, 2021

Efanesoctocog alfa, a novel and investigational factor VIII therapy independent of von Willebrand Factor, is designed to provide near-normal factor activity levels for the majority of the week in a once-weekly prophylactic treatment regimen. In 2019, Sobi’s revenues amounted to SEK 14.2 billion.

FDA 52

FDA Treatment Therapies International 52

Drug Target Review

NOVEMBER 8, 2023

Cell therapies hold immense promise for revolutionising the treatment of some of the most challenging diseases, but several technological and logistical hurdles stand in the way of unlocking their full potential. By harnessing the fundamental biology of T cells, Senza5 can be used to improve the efficacy of almost any CAR-T therapy.

Therapies 116

Therapies Engineering Treatment Molecular Biology 116

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

NOVEMBER 13, 2023

This commitment to the Gerstner Center for Cancer Diagnostics includes $10 million for current support for the Center and $10 million in endowed funding, adding to the $15 million originally committed by Gerstner and an initial endowment of $10 million from the Eli & Edythe Broad Foundation to establish the Center in 2019.

DNA 116

DNA Medical Schools Treatment Therapies 116

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.

DNA 145

DNA Engineering RNA Laboratories 145

The Pharma Data

MARCH 2, 2021

Boehringer Ingelheim and Gubra today announced a new research and licensing agreement focused on the identification and validation of targets and innovative peptide compounds for the treatment of obesity. Senior Vice President and Global Head of Cardiometabolic Diseases Research, Boehringer Ingelheim. population will have obesity.

Treatment 52

Treatment Bioinformatics Clinical Development Therapies 52

The Pharma Data

APRIL 7, 2021

Data from the pivotal Phase 3 CARDINAL study demonstrated sutimlimab inhibited C1-activated hemolysis (abnormal destruction of healthy red blood cells) within one week of treatment and had a sustained treatment effect over the course of the study. g/dL at treatment assessment timepoint. Europe, and Japan. 2 g/dL by week 3.

Disease 52

Disease Trials Treatment Therapies 52

Drug Target Review

OCTOBER 4, 2023

Antibody-drug conjugates (ADCs) have been a groundbreaking approach to cancer treatment with their ability to deliver cytotoxic drugs directly to diseased cells while sparing healthy tissues. Ultimately, flexibility in dual-payload ADC development means better treatments could reach patients sooner.

Small Molecule 126

Small Molecule Drugs Immune Response Targeted Protein Degradation 126

The Pharma Data

JANUARY 26, 2021

Novel chronic kidney disease (CKD) target from the partnership enters AstraZeneca’s portfolio. In 2019, BenevolentAI and AstraZeneca entered a strategic collaboration aiming to discover new drugs for CKD and idiopathic pulmonary fibrosis (IPF). LONDON , Jan. LONDON , Jan. Meta description.

Disease 52

Disease Clinical Development Treatment Drug Development 52

The Pharma Data

JANUARY 14, 2021

Department of Health and Human Services’ (HHS) decision to effectively remove the “X-waiver” requirement for physicians, expanding their ability to utilize medication-assisted treatment (MAT) for patients struggling with opioid use disorder (OUD). Added Dr. Rosenberg, “This is a great day for our patients.

Treatment 52

Treatment Licensing Licensing Hospitals 52

Drug Target Review

DECEMBER 4, 2023

Tailoring medical care to a patient’s unique genetic makeup, alongside the close analysis of disease progression, leads to more effective treatments, reduced side effects and faster diagnoses. What’s more, establishing the clinical utility of genetic markers and treatment strategies is a time-consuming and resource-intensive process.

Treatment 105

Treatment Science DNA Doctors 105

The Pharma Data

JANUARY 17, 2021

n-Lorem Foundation has taken on a challenge that many nations consider too great: treating patients with ultra-rare diseases (which affect 30 or fewer people) for free, for life. n-Lorem selects patients based on their genetic mutation and the organ affected rather than their specific diseases. “We they can help many patients.”.

Disease 52

Disease Clinical Trials Small Molecule Treatment 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. Understanding how they do this may inspire treatment strategies for the wider patient population. Quiroz, Ph.D.,

Disease 97

Disease Research Therapies DNA 97

The Pharma Data

SEPTEMBER 27, 2021

AbbVie Submits Regulatory Application to FDA for Risankizumab-rzaa (SKYRIZI®) for the Treatment of Patients 16 Years and Older with Moderate to Severe regional enteritis. 9-11 it’s a progressive disease, meaning it can worsen over time.10,11 9-11 it’s a progressive disease, meaning it can worsen over time.10,11

Treatment 52

Treatment FDA Therapies Disease 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.”

Disease 98

Disease Bioinformatics Medical Schools Research 98

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.

Small Molecule 62

Small Molecule DNA Disease Trials 62

Broad Institute

JANUARY 30, 2024

In others, an infection can cause the immune system to spin out of control, leading to sepsis — organ dysfunction that lacks effective treatments and is often fatal. An analysis estimated that nearly 5 million deaths in 2019 were associated with bacterial resistance to antimicrobial medicines, including 1.27

Hospitals 137

Hospitals Virus Medical Schools Science 137

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

The Pharma Data

JULY 22, 2021

NOTE: ADUHELM TM (aducanumab-avwa)injection 100 mg/mL solutionis indicated for the treatment of Alzheimer’s disease. Treatment with ADUHELM should be initiated in patients with mild cognitive impairment or mild dementia stage of disease, the population in which treatment was initiated in clinical trials.

Disease 52

Disease Research Clinical Trials FDA 52

Drug Target Review

NOVEMBER 17, 2023

They do not induce ‘graft versus host’ disease when transplanted for allogeneic therapy and there appears little sign of immune rejection. Only three of the CAR-NK studies were for the treatment of solid tumours. NK cells can be readily extracted from umbilical cord blood or peripheral blood of adult donors.

Therapies 118

Therapies Clinical Trials Cell Biology Trials 118