XTalks

JUNE 12, 2024

It leverages various patient-specific factors, including genetics, age, weight, organ function and even lifestyle, to determine the most appropriate dosage for each individual. Her career spans pediatric clinical pharmacy, clinical research and pharmacogenomics with leadership roles at University of Chicago Medical Center and PipelineRx.

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Life Science Pharmacy Doctors Doctor 97

Scienmag

JULY 4, 2022

Medical and life science researchers will benefit from the most comprehensive atlas yet of genetic data on zebrafish, newly published research suggests. The atlas will help researchers to better study conditions from various types of cancer (e.g. skin cancer), heart disease, and neurodegeneration.

Genetics 61

Genetics Research Life Science 61

XTalks

JANUARY 6, 2025

CF is a progressive genetic disease caused by defective CFTR proteins, which are crucial for regulating salt and water movement in cells. Vertex remains committed to advancing CF research, with plans to expand the impact of their CFTR modulators and explore new genetic mutations.

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Gene Therapy Protein Genetic Disease Gene 105

XTalks

APRIL 17, 2025

Approximately 80% of these diseases have a genetic basis , and many manifest in childhood , often with significant challenges in achieving a timely and accurate diagnosis. This variability not only poses challenges for diagnosis but also stresses the need for targeted research and innovative therapeutic approaches.

Clinical Research 52

Clinical Research Research Clinical Trials Clinical Trials 52

XTalks

FEBRUARY 4, 2025

The company plans to use these funds to advance its groundbreaking research in small molecule precision medicines, targeting renal, cardiovascular and metabolic diseases, including obesity. For complex diseases like chronic kidney disease (CKD), where genetics play a significant role, such a platform holds promise.

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Clinical Trials Clinical Trials Trials Genetics 66

XTalks

APRIL 13, 2022

To learn more about new Parkinson’s disease research, Ayesha discussed a new study by researchers at Rush University Medical Center that shows statins may help decrease the risk of parkinsonism in older adults. Genedrive’s Genetic Test Can Prevent Hearing Loss in Babies Through Personalized Treatment.

Life Science 52

Life Science Genetics Research Development 52

World of DTC Marketing

JANUARY 24, 2022

That investment, equal to the National Institutes of Health’s annual spending on medical research, would seem like great news for a world waiting for help with a pandemic. A group of early-stage life science investors and biotech CEOs joined together with a prominent patient advocate to speak out against H.R.3,

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Life Science Drugs Development Genetic Disease 225

Pharmaceutical Technology

MAY 4, 2023

Forge Biologics and global life sciences company Labcorp have announced a strategic adeno-associated virus (AAV) gene therapy development and manufacturing collaboration.

Gene Therapy 256

Gene Therapy Gene Development Manufacturing 256

XTalks

AUGUST 7, 2024

Lawreen is a seasoned biotech and life science professional with over 25 years of experience. She began her career as a research scientist, working for more than eight years in this role before transitioning into product management and marketing. Subscribe to the Xtalks Life Science Podcast to never miss a new episode.

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Life Science Scientist Production Research 52

XTalks

FEBRUARY 28, 2024

Ayesha spoke about how the Rare Disease Day campaign helps increase awareness about rare diseases, promotes research and development, supports advocacy for policies supporting patients and their families and brings together a global rare disease community. Subscribe to the Xtalks Life Science Podcast to never miss a new episode.

Life Science 52

Life Science Research Genetics Drugs 52

XTalks

DECEMBER 9, 2024

This initiative is particularly impactful for rare diseases and genetic disorders, where traditional endpoints may be infeasible. Programs like the Rare Disease Endpoint Advancement Pilot encourage collaboration with the FDA to establish novel surrogate endpoints.

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Clinical Trials Clinical Trials Compliance Trials 105

pharmaphorum

FEBRUARY 26, 2021

For Rare Disease Day, RBW Consulting’s Emma Thorp discusses why the consultancy is taking revenue earned from its rare disease clients and using it to fund research into the mental health impact of a rare disease diagnosis. Issues like this, and experiences like mine, are why RBW is funding a partnership with Rare Disease Research Partners.

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XTalks

FEBRUARY 2, 2022

The Israeli Society of Gene and Cell Therapy (ISGCT) is hosting its upcoming annual meeting in-person this year to encourage networking and the sharing of information about genetic and cellular therapies. The aim of the ISGCT is to foster and maintain strong scientific communities in this field of research.

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Gene Research Genetics Clinical Trials 98

Pharmaceutical Technology

JANUARY 12, 2023

Solid Biosciences has entered a strategic research collaboration with Phlox Therapeutics to expedite the development of new therapies for rare cardiac diseases. Under the research partnership deal, the companies will develop new precision genetic medicines for a severe form of genetic dilated cardiomyopathy (DCM).

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RNA Gene Therapy Genetics Genetic Disease 130

XTalks

JANUARY 26, 2024

Technological advancements are revolutionizing cancer research and therapies. By editing these genes, researchers can effectively neutralize their cancer-promoting effects. By tailoring treatments based on an individual’s genetic makeup, it may allow for more effective and targeted therapies.

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Research Gene Editing Gene Genetics 118

Pharmaceutical Technology

NOVEMBER 29, 2022

For several decades, researchers have been investigating the role of ketamine in treating depression. PharmaTher also has an agreement in place with Case Western Reserve University to develop ketamine as a treatment for Rett syndrome, a rare genetic neurological disorder. Depression research spurs efforts for other conditions.

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XTalks

MARCH 2, 2022

The editorial team discussed the encouraging growth seen in recent years in rare disease clinical research in the life science industries, providing hope for improved diagnosis, care and treatments for patients with rare diseases. Subscribe to the Xtalks Life Science Podcast to never miss a new episode.

Life Science 52

Life Science Clinical Trials Clinical Trials Trials 52

XTalks

FEBRUARY 4, 2021

Generation of strong research dataset cohorts must begin with high-quality clinical samples. Moreover, biobanks are no longer passive biorepositories for accrual of samples and serve a more utilitarian function in identifying and coordinating specific research cohorts for longitudinal and prospective studies.

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Research Biobanking Drugs Genome 98

XTalks

NOVEMBER 14, 2024

This therapy, the first of its kind to be administered directly into the brain, addresses a rare genetic disorder that severely affects motor function and dopamine production.

Gene Therapy 105

Gene Therapy FDA Approval Gene Clinical Trials 105

XTalks

SEPTEMBER 22, 2022

In this episode, Ayesha shared new research about how scientists at the Francis Crick Institute and University College London have found a mechanism of action to explain how air pollution may cause lung cancer in non-smokers. can act on existing genetic mutations to trigger inflammation and the development of cancer.

Life Science 52

Life Science Scientist Development Genetics 52

pharmaphorum

SEPTEMBER 28, 2022

Martin Mendoza, PhD, director of health equity and health science policy for the All of Us Research Program at the National Institutes of Health (NIH), tells pharmaphorum about the programme and how it will accelerate research for the benefit of all patients. Gathering data.

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Research Medicine Clinical Trials Clinical Trials 98

XTalks

SEPTEMBER 9, 2024

Diversity, equity and inclusion (DEI) have become important elements in the landscape of clinical research. Xtalks spoke with Stacy Weil, DHI, Senior Vice President, Business Operations and Patient Centricity, Premier Research, to gain insights into incorporating DEI practices and plans in clinical research.

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Clinical Research Research Clinical Trials Clinical Trials 101

XTalks

APRIL 3, 2025

Eli Lilly shared new Phase II trial data showing that its experimental siRNA therapy, lepodisiran, leads to reductions in levels of lipoprotein(a) (Lp(a)) a genetically inherited risk factor for heart disease in adults with elevated levels of the marker. High Lp(a) levels can double or even triple the risk of a heart attack.

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Trials Clinical Trials Clinical Trials Genetics 59

XTalks

NOVEMBER 15, 2024

Drug resistance in DEEs often stems from genetic and molecular factors unique to each syndrome, limiting standard treatments’ effectiveness. This extension aims to provide insights into long-term safety, addressing a common gap in epilepsy research. She noted that DEEp OCEAN is a step towards inclusivity in clinical research.

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Trials Clinical Trials Clinical Trials Genetics 97

XTalks

APRIL 8, 2025

There are over 10,000 rare diseases affecting an estimated 300 million people worldwide where 80% are genetic , 95% lack approved treatments and nearly half begin in childhood. Cross-border enrollment captures a wider range of genetic backgrounds, environmental influences, and cultural perspectives.

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Clinical Trials Clinical Trials Trials Gene Therapy 88

XTalks

DECEMBER 31, 2024

points as assessed by the Medical Research Council (MRC) sum score. Severe GBS patients treated with a single dose of imlifidase (0.25 By the first week, 37 percent of patients were able to walk independently. The mean improvement in muscle strength was 10.7 By eight weeks, 67 percent of patients were able to walk independently, 40.7

Life Science 52

Life Science Clinical Trials Clinical Trials Antibody 52

XTalks

DECEMBER 12, 2024

In clinical research, this includes designing trials that elevate the patient voice, reduce barriers to participation, and represent the diverse populations who will ultimately use these medical products. This distrust is further compounded by a lack of visible accountability and representation in the research process.

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Clinical Trials Clinical Trials Clinical Research Trials 59

pharmaphorum

OCTOBER 7, 2020

Originally discovered in bacteria as a defense mechanism against pathogens, CRISP/Cas9 has made it possible to make exquisitely detailed and precise alterations to DNA sequences on demand, and as a tool for molecular biology has already transformed research into diseases and drug discovery. What started as a curiosity?driven,

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Gene Editing Bacteria DNA Gene 105

Medical Xpress

DECEMBER 13, 2024

Researchers at Nano Life Science Institute (WPI-NanoLSI), Kanazawa University, have discovered how a protein called lamin A helps repair the protective barrier around a cell's DNA.

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DNA Genetics Life Science Protein 52

XTalks

MARCH 17, 2025

Some research suggests that optimized microfluidic devices can isolate rare CTCs with recovery rates exceeding those of conventional methods sometimes reaching over 90% under ideal conditions.

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Production Manufacturing DNA Genetics 71

XTalks

MARCH 24, 2021

A new study published in the journal Science Advances has reported the identification of 50 previously unknown genes involved in the determination of human eye color variation. The study was led by a team of researchers at King’s College London and Erasmus University Medical Center Rotterdam. The researchers say that collectively, 53.2

Genetics 59

Genetics Gene Genome Genomics 59

pharmaphorum

DECEMBER 14, 2022

As many strive to bring about transformation in healthcare, Angelini Ventures takes a new approach to investments in digital health and life sciences companies that drive healthcare innovation and improve patient experiences. It aims to shape companies in the digital healthcare, connected medical devices, and life sciences spaces.

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Life Science Development Business Development Genetics 105

XTalks

FEBRUARY 18, 2021

The study , published in the journal Science of the Total Environment , offers the first lines of evidence for how climate change could have played a direct role in the appearance of the COVID-19 pandemic-causing virus. The research also highlights the potential mechanism involved in the phenomenon. Comparative Analysis and Mapping.

Research 98

Research Engineer Genetics Life Science 98

XTalks

MAY 17, 2023

The FNIH will focus on advancing clinical trials for the eight diseases through gene therapy research partnerships with the goal of accelerating the path of new therapies to clinical trials. Of the more than 10,000 rare diseases, more than 80 percent are caused by genetic defects.

Gene Therapy 98

Gene Therapy Clinical Trials Clinical Trials Gene 98

XTalks

MARCH 7, 2024

Other issues include medical systems that can often be dismissive of women’s health concerns or experiences, and historically less research on women’s health, which together, can lead to late diagnoses or misdiagnoses. Through this, researchers can pinpoint breast cancer cell types and their phenotypic states.

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XTalks

MAY 20, 2022

Do you possess laboratory skills, an analytical mindset and a penchant for research? A clinical scientist conducts research to help prevent illness and develops techniques to support patient diagnosis and treatment. They will often find themselves working in a healthcare or pharmaceutical field that supports human health research.

Scientist 105

Scientist Life Science Clinical Research Doctors 105

pharmaphorum

FEBRUARY 17, 2021

One group excelling in this space is the Genetic Alliance. Their work ranges from developing new services in partnership with the National Health Service (NHS) to working with the All Party Parliamentary Group on Rare, Genetic and Undiagnosed Conditions, and the Department of Health on the UK Strategy for Rare Diseases.

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