biobide

FEBRUARY 2, 2024

Pediatric cancers exhibit differences at the genetic level compared to the same form of adult disease, which may influence the selected treatment. Children and adolescents lack robust preclinical models to replicate the pathologies and provide precise and targeted therapies.

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Advarra

MARCH 10, 2023

Having a one in four chance of developing sickle cell disease (SCD) is uniquely, and overwhelmingly, familiar to the African American population. Considered a rare disease, research in finding better therapies – and possibly even a cure – for SCD may be competing for funding with conditions impacting broader populations.

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Disease Clinical Trials Doctors Trials 98

Drug Discovery World

JUNE 21, 2024

Allogene Therapeutics and Foresight Diagnostics have announced the initiation of a Phase II trial evaluating the use of cemacabtagene ansegedleucel (cema-cel) as part of the first line treatment regimen for newly diagnosed large B-cell lymphoma (LBCL) patients who are likely to relapse.

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Drug Discovery Today

JANUARY 13, 2021

Mission: Cure is a coalition of patients, doctors, researchers and entrepreneurs who are committed to advancing new therapies and cures for currently untreatable diseases. Their primary disease focus is on chronic pancreatitis, a rare inflammatory condition that affects over a million people across the globe.

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Treatment Doctors Disease Therapies 113

Drug Target Review

JUNE 3, 2024

How does COUR Pharmaceuticals’ immune-modifying nanoparticle platform differ from traditional approaches to treating immune-mediated diseases? Essentially, our CNPs reprogramme the immune system by restoring balance and returning the body to a homeostatic state, offering a precise approach to treating immune-mediated diseases.

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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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Drug Discovery World

MAY 20, 2024

GLP-1 receptor agonists like Ozempic (semaglutide) are a class of incretin mimetic medicines for the treatment of type 2 diabetes and obesity. GlobalData’s report predicts that GLP-1 receptor agonists will dominate the metabolic diseases space in the next 10 years.

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Drug Target Review

SEPTEMBER 1, 2023

Could you explain the fundamental distinction between autologous and allogeneic cell therapies and how they differ in terms of cell sourcing and application? Autologous immune cell therapy involves the grafting of a patient’s own immune cells. Then, cells are infused back to that same patient.” There are pros and cons to each.

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Drug Target Review

JANUARY 12, 2024

How does the integration of Real-World Data (RWD) with genomic biomarker data contribute to a more comprehensive understanding of disease progression and treatment response? This one size fits all approach to drug prescribing still pervades in mostly all therapy areas except oncology. Most drugs do not work in all people.

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Doctors Clinical Trials Treatment Therapies 109

FDA Law Blog: Drug Discovery

MARCH 13, 2023

Who better than people living with a condition to inform drug companies, physicians, academics, and the FDA on what it is like to live with their condition, what symptoms most impact their lives, what goes into their decision about whether to participate in a clinical trial, and what kind of treatment effects would be most meaningful to them?

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Drug Discovery World

NOVEMBER 13, 2023

Nipocalimab, according to Abouzahr, is currently the only FcRn being studied in all three segments of autoantibody-driven disease. She says: “At Janssen, we’re fortunate that we’re building on a leadership that we’ve created over the last couple of decades of treating immune-mediated diseases.

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PLOS: DNA Science

OCTOBER 12, 2023

In the final chapter of my 2012 book The Forever Fix: Gene Therapy and the Boy Who Saved It , I predicted that the technology would soon expand well beyond the rare disease world. Gene therapy clearly hasn’t had a major impact on health care, offering extremely expensive treatments for a few individuals with rare diseases.

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Olympian Clinical Research

NOVEMBER 9, 2022

Although this disease is estimated to affect less than 50,000 Americans, it may be life-threatening, especially for people who are already in poor health. Therefore, if you or a loved one is dealing with this rare condition, it’s essential to educate yourself about the condition and its potential treatments. Radiation therapy.

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KIF1A

JUNE 4, 2024

“We really hope that someday there will be a treatment that can help stop the progression of the symptoms, and give Elena and us more years to enjoy together.” Hundreds of doctor appointments and uncountable exams, second and third opinions, but nothing made sense. The day we received the KAND diagnosis the world stopped.

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The Pharma Data

AUGUST 22, 2021

In the early days of the COVID-19 pandemic, doctors in Wuhan noticed something surprising. “There’s often a phenomenon that doctors report anecdotally, or that’s mentioned in passing in a particular research paper, and that provides a clue – a hook,” he said. The next challenge was to figure out why.

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Drug Discovery World

JANUARY 22, 2024

For the patient as well as for doctors, this means creating more time to make decisions, as the company believes: “Patients – and their quality of life – shouldn’t have to wait for a broken system to provide an answer six months too late.

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Drug Target Review

FEBRUARY 29, 2024

During my academic career, I had the opportunity to examine the underlying causes of neurodegenerative diseases like Parkinson’s disease and develop novel animal models to investigate potential disease-modifying therapeutics. AskBio) I continue to work to bring innovative gene therapies to patients in need.

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Drug Discovery World

APRIL 3, 2023

DS: What motivated you and Dr David Bunka, CTO, to create Aptamer and did you imagine you would one day be operating on such a global scale?    AT : We initially met at the University of Leeds, where David was a post-doctoral researcher studying aptamers, and I was completing my PhD. DS: The company now has clients across big pharma.

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Advarra

JUNE 27, 2023

A clinical trial investigating a drug or therapy is typically divided into four phases: Phase I, Phase II, Phase III, and Phase IV. Each phase has a different purpose in testing the treatment’s safety and efficacy. Currently incurable diseases would remain so, without any improvements made to existing drugs and therapies.

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The Pharma Data

NOVEMBER 20, 2020

20, 2020 — Hormone therapy can be a lifesaver for men with prostate cancer , but it also appears to put some at increased risk of heart problems, a new study reports. At the start of the study, 4 out of 5 men had two or more risk factors for heart disease , the study says. FRIDAY, Nov.

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Alta Sciences

JUNE 27, 2023

Cancer Treatments, Clinical Trials, and the Motivation to Innovate nbartlett Tue, 06/27/2023 - 20:58 Getting to the Heart of Science with Michelle Newby At the heart of science are stories—stories of courage, challenges, successes, lessons, and people. Q: What was your cancer diagnosis and was treatment options were you given?

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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.

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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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Drug Discovery World

JUNE 5, 2023

Showcase agenda Throughout the day, there were a number of presentations on the exhibition floor which provided company updates and showcased a number of exciting results and projects, highlighting both how far we have come in the treatment of disease, as well as how far there is still to go.

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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.

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The Pharma Data

JANUARY 20, 2021

Food and Drug Administration (FDA) has accepted the company’s supplemental New Drug Application (sNDA) and granted Priority Review for Esbriet ® (pirfenidone) for the treatment of unclassifiable interstitial lung disease (UILD). In 2020, the FDA granted Orphan Drug Designation and Breakthrough Therapy Designation to Esbriet for UILD.

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The Pharma Data

SEPTEMBER 23, 2020

Even with the family hardships, seeing her sister battle her illness gave Lisa the strength to face this new challenge head-on and take a leading role in her disease. I challenged my doctor quite often, always asking questions, ‘What’s next? I had to rely on the doctor for all my answers.”. What can you do for me?

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Drug Target Review

FEBRUARY 26, 2024

Regarding therapy resistant melanomas, why is the alteration of the epigenetic landscape considered significant, and what has been lacking in terms of validated targetable epigenetic mechanisms? What is remarkable here is that we show that the addition of corin to BRAFi-resistant tumour cells resensitises them to BRAFi therapies.

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Drug Discovery World

FEBRUARY 1, 2023

There are more than 1300 clinical trials on cannabis underway and companies focused on the cultivation and manufacturing of cannabis are also educating doctors and patients about its potential pharmaceutical use. Meanwhile, the Pfizer purchase of Arena Pharmaceuticals, highlights cannabinoid-based bowel disease treatments as a promising area.

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Drug Target Review

JULY 3, 2024

With a predominantly industrial background, I attended an engineering school before embarking on a doctoral thesis in science. It was the focus of my doctoral thesis, eventually leading to the co-founding of a startup. I persevered with the support of mentors as I was determined to pursue my passion.

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The Pharma Data

JANUARY 15, 2021

Food and Drug Administration (FDA) approval of Darzalex Faspro ® (daratumumab and hyaluronidase-fihj), a subcutaneous formulation of daratumumab, in combination with bortezomib, cyclophosphamide and dexamethasone (D-VCd) for the treatment of adult patients with newly diagnosed light chain (AL) amyloidosis.[1]

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The Pharma Data

JANUARY 17, 2021

The treatment could be administered in primary care centers to people who test positive for COVID-19, avoiding hospitalization due to the progression of the disease and complementing the vaccine in the early phase after vaccination. BARCELONA, Spain , Jan. 18, 2021 /PRNewswire/ — Grifols (MCE: GRF, MCE: GRF.P,

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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.

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KIF1A

APRIL 27, 2024

By ANDREA SWINDALL, First Published: 8:34 AM PDT, April 6, 2024 KIF1A Associated Neurological Disorder is a rare severe neurodegenerative disease caused by mutations in the KIF1A gene, Amanda Burrett says. We had her one-year checkup, and the doctor was concerned about her lack of awareness to the environment around her. Amanda says.

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The Pharma Data

SEPTEMBER 10, 2021

More than 20 abstracts across disease states, including Parkinson’s disease, spasticity and cervical dystonia, will be presented. “Our research presented at MDS 2021 builds upon our expertise in neuroscience and reinforces our mission to advance the standards of care for people living with these debilitating diseases.”

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The Pharma Data

NOVEMBER 4, 2020

New data will also be presented that show the safety and efficacy of ULTOMIRIS when used concomitantly with immunosuppressive therapy (IST) in patients with PNH. aHUS is an ultra-rare disease that can cause progressive injury to vital organs, primarily the kidneys, via damage to the walls of blood vessels and blood clots.

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The Pharma Data

JULY 21, 2021

A BTD is intended to expedite the development and review of medications to treat a serious medical condition and is granted when preliminary clinical evidence indicates the investigational therapy may demonstrate substantial improvement over existing therapies. PRNewswire/ — AbbVie (NYSE: ABBV) announced today that the U.S.

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