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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Therapies Disease Clinical Trials Science 111

KIF1A

APRIL 6, 2024

Each week, Dr. Dylan Verden of KIF1A.ORG summarizes newly published KIF1A-related research and highlights progress in rare disease research and therapeutic development. As our genetic analyses advance, studies like this highlight the value in reassessing established disease populations. She graduated from UC San Diego with a B.S.

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DNA Medical Schools Therapies Research 111

Drug Target Review

DECEMBER 16, 2024

Getting into medical school was very competitive with thousands of candidates vying for a handful of positions, but I was fortunate enough to succeed. My inclination toward biological sciences led me to medical school to pursue a future in STEM. I opted for medicine due to my love for biological sciences.

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Medical Schools Clinical Research Drug Development Trials 52

The Pharma Data

JANUARY 25, 2021

25, 2021 — Recommendations are presented for the diagnosis and management of von Willebrand disease (VWD) in two clinical guidelines issued by the American Society of Hematology, International Society on Thrombosis and Haemostasis, National Hemophilia Foundation, and World Federation of Hemophilia and published online Jan. MONDAY, Jan.

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Drug Target Review

AUGUST 29, 2024

What potential advantages does the CD5 modulation strategy offer over traditional CAR-T therapies? MR : Chimeric antigen receptor T-cell (CAR-T) therapy is very effective in treating patients with B-cell lymphoma, leukemia, and multiple myeloma, where we have six FDA-approved drugs.

Therapies 59

Therapies Treatment Engineering Molecular Biology 59

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

Broad Institute

AUGUST 19, 2024

This study is an important example of how understanding core metabolic requirements and functions of key bacteria can lead directly to new therapies that allow us to modify the microbiome for improved health,” said co-senior author Seth Bloom, an infectious diseases instructor at the Massachusetts General Hospital.

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Hospitals Treatment Medical Schools Science 121

Broad Institute

NOVEMBER 13, 2023

The initial focus of the Gerstner Center is to develop an ultrasensitive liquid biopsy test to detect minimal residual disease (MRD) — the presence of tumor cells in the body after treatment. Doing so would guide treatment decisions, such as whether to continue therapy. "We

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DNA Medical Schools Treatment Therapies 116

Broad Institute

JUNE 30, 2023

Common genetic variants associated with cardiometabolic disease can produce phenotype changes of such small effect that they can be difficult to characterize. Common genetic variants associated with cardiometabolic disease can produce phenotype changes of such small effect that they can be difficult to characterize.

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The Pharma Data

DECEMBER 15, 2020

A healthy immune system defends the body against disease and other conditions. Autoimmune disease impacts different parts of the body, weakening functionality. Researchers are aware of more than 80 diseases that occur when the immune system attacks the body’s own organs, tissues and cells. It may be life-threatening.

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Disease Clinical Trials Small Molecule Trials 52

The Pharma Data

JUNE 2, 2022

Novartis today announced a partnership with the American Society of Hematology (ASH) to provide six additional African nations with technology that is already being used in Ghana to document and share the diagnosis of babies with sickle cell disease.

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Disease Medical Schools Treatment Therapies 52

Nvidia Developer: Drug Discovery

JULY 14, 2022

Drug discovery startup Insilico Medicine—alongside researchers from Harvard Medical School, Johns Hopkins School of Medicine, the Mayo Clinic, and. ALS is a debilitating disease. Today, patients typically face an average life expectancy of between two and five years after symptom onset. This is only the beginning.”

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Medical Schools Clinical Trials FDA Approval Engineering 52

The Pharma Data

SEPTEMBER 18, 2020

Promising Phase 2 data has been released at the European Society for Medical Oncology (ESMO) Virtual Congress 2020 for Sanofi and Regeneron’s jointly-developed PD-1 inhibitor Libtayo (cemiplimab) in the treatment of locally advanced basal cell carcinoma (BCC) in patients for whom hedgehog inhibitor (HHI) therapy is not applicable.

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Medical Schools Trials Therapies Hospitals 52

The Pharma Data

JUNE 2, 2023

Zouboulis, President of the European Hidradenitis Suppurativa Foundation, Director of the Departments of Dermatology, Venereology, Allergology and Immunology, Städtisches Klinikum Dessau, and Founding Professor of Dermatology and Venereology at the Brandenburg Medical School, Germany.

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Medical Schools Treatment Disease Therapies 52

The Pharma Data

JANUARY 17, 2021

Optometrists provide comprehensive eye care, including evaluations for glasses and contact lenses and common eye diseases. Ophthalmologists are physicians who have completed four years of medical school and four years of residency training. They can also treat acute eye problems such as pink eye or sties.

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Medical Schools Doctors Treatment Disease 52

Broad Institute

JULY 26, 2022

By Stephanie Dutchen, Harvard Medical School Office of Communications and External Relations July 26, 2022 Credit: Susanna Hamilton, Broad Communications From immunity to metabolism to mental health, it seems like the gut microbiome has been linked to every aspect of human health and disease. muciniphila ’s cell membrane.

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Research Immune Response Medical Schools Disease 52

NIH Director's Blog: Drug Discovery

DECEMBER 9, 2014

Tanzi, Massachusetts General Hospital/ Harvard Medical School Researchers want desperately to develop treatments to help the more than 5 million Americans with Alzheimer’s disease and the millions more at risk. The answer appears to lie in their decision to add a third dimension to their disease model.

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Drugs Disease Hospitals Medical Schools 52

Drug Target Review

JULY 1, 2024

As a non-NSAID, it reduces adverse effects associated with these pain relief drugs, including kidney injury, GI gastritis/ulceration and blood pressure elevation, which can affect patients with cardiovascular disease and the elderly. was founded in 2016 to develop safer, non-opioid therapies for acute and chronic pain.

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Treatment Clinical Trials Therapies Molecular Biology 59

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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Therapies Molecular Biology Medical Schools Regulations 59

NIH Director's Blog: Drug Development

APRIL 2, 2020

With so much information swirling around these days about the coronavirus disease 2019 (COVID-19) pandemic, it would be easy to miss one of the most interesting and significant basic science reports of the past few weeks. Credit: Adapted from Yan R., Science, 2020. The team, led by Scott D.

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Drug Development Drugs Science Medical Schools 52

The Pharma Data

MARCH 13, 2021

Further, prespecified exploratory analyses showed donanemab slowed the accumulation of tau across key brain regions in patients affected by Alzheimer’s disease. “This is the first late-stage study in Alzheimer’s disease to meet its primary endpoint at the primary analysis. Apostolova, M.D.,

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Disease Trials Treatment Medical Schools 40

The Pharma Data

NOVEMBER 22, 2020

At the moment, CRISPR Cas-9 is only utilized for rare diseases on cells that have been removed from the body. The product is the most advanced gene-editing approach in development for transfusion-dependent beta thalassemia (TDT) and severe sickle cell disease. Cas-9 represents the Cas9 protein, an enzyme that cuts foreign DNA.

DNA 52

DNA Medical Schools Treatment Science 52

The Pharma Data

DECEMBER 13, 2020

which develops genome editing technologies to accelerate drug discovery and develop novel therapeutics for a broad range of diseases, today announced the appointment of Bo Zhang, Ph.D., Prior to joining EdiGene, he was Vice President of KLUS Pharma and focused on cell therapy and new technologies. as Head of Business Development.

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Medical Schools Clinical Development Therapies RNA 40

On Medicine

APRIL 6, 2023

I have a background in Biomedical Engineering prior to medical school and a particular interest in global health and healthcare in resource-limited settings. The chronic cardiovascular diseases are frequently insidious but pervasive and progressive, and early subclinical changes are difficult to identify in children.

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Medical Schools Hospitals Treatment International 52

The Pharma Data

JANUARY 26, 2021

Dr. Short brings to VarmX extensive clinical development expertise, with a particular focus on rare diseases and gene therapy for haematological disorders. Further, he advanced gene therapy clinical programmes in haemophilia B and Fabry’s disease. Dr. Short received his Bachelor of Medicine (MBBS) degree from St.

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Clinical Development Medical Schools Therapies Pharmaceuticals 40

Broad Institute

JUNE 21, 2023

Common genetic variants associated with cardiometabolic disease can produce phenotype changes of such small effect that they can be difficult to characterize. Common genetic variants associated with cardiometabolic disease can produce phenotype changes of such small effect that they can be difficult to characterize.

Small Molecule 52

Small Molecule Cell Based Assays Medical Schools Disease 52

The Pharma Data

JUNE 7, 2023

These longer-term results show the durable overall survival benefit of sacituzumab govitecan over traditional chemotherapy in pre-treated HR+/HER2- metastatic breast cancer,” said Sara Tolaney, MD, MPH, Chief of the Division of Breast Oncology at the Dana-Farber Cancer Institute, and Associate Professor of Medicine, Harvard Medical School. “At

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Medical Schools Therapies Treatment Clinical Development 40

Broad Institute

AUGUST 4, 2022

By Tracy Hampton August 4, 2022 Credit: National Institutes of Allergy and Infectious Diseases, National Institutes of Health Colorized scanning electron micrograph of a B cell lymphocyte from a human donor.

Bioinformatics 52

Bioinformatics Medical Schools Hospitals Treatment 52

The Pharma Data

JANUARY 25, 2021

NeoVax is being studied as an additional therapy for other cancers, including later-stage ovarian and kidney cancers. . “The vaccine can induce a durable immune system response that is well-tolerated.” ” In theory, Ott said, personalized vaccines could be used for a range of cancers. SOURCES: Patrick A. 21, 2021, online.

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Vaccine Immune Response Medical Schools Clinical Trials 52

KIF1A

MAY 31, 2024

Chung serves as the Mary Ellen Avery Professor at Harvard Medical School (HMS), and President of the Children’s Hospital Pediatric Associates. ASCEND Online and KOALA In-Person Natural History Study Updates Understanding KAND and the progression of the disease informs our path to treatment.

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Treatment Small Molecule International Research 52

The Pharma Data

DECEMBER 28, 2020

Scopus is a biopharmaceutical company whose lead drug candidate is a novel, targeted immuno-oncology gene therapy for the treatment of multiple cancers. Dr. Hofstein completed his postdoctoral training and research in the Department of Neurobiology at Harvard Medical School. Hofstein received his B.Sc.

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Drug Target Review

AUGUST 28, 2024

What are the main challenges currently faced in the treatment of chronic liver diseases, and how does Resolution Therapeutics aim to address these challenges? Once a patient develops advanced cirrhosis/end-stage liver disease there are no specific therapies to significantly avoid major decompensations and death in the next few years.

Therapies 116

Therapies Disease Engineering Medical Schools 116

The Pharma Data

DECEMBER 5, 2020

5, 2020 — A pair of new gene therapies promise a potentially lasting cure for sickle cell disease by subtly altering the genetic information in patients’ bone marrow cells, researchers report. Lewis Hsu, chief medical officer of the Sickle Cell Disease Association of America. SATURDAY, Dec.

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Therapies Disease Medical Schools Virus 52

Broad Institute

DECEMBER 6, 2023

Cytokines are also targeted by drugs for many diseases such as rheumatoid arthritis, COVID-19, and cancer, but until now, scientists haven’t had a comprehensive view of how different immune cells respond to different cytokines because the immune system is so complex. For many immune-mediated diseases, there's no cure or treatment.

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Immune Response RNA Disease Medical Schools 138

Broad Institute

MAY 2, 2024

If we can understand how those processes work, we can better develop drugs to target these multifaceted diseases. With rapid autopsies, you can look at a cancer’s life and see how it developed from a primary to metastatic disease. In undergrad, I thought about going to medical school, but I didn’t want to work in a hospital.

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Science Medical Schools Disease Hospitals 124

Drug Target Review

JANUARY 15, 2024

This produces a great advantage in infectious disease, as it becomes difficult for a virus to adapt and spread from person to person, because each person’s HLA array is genetically distinct. Engineering soluble T-cell receptors for therapy. Cancer Therapy With TCR‑Engineered T Cells. The FEBS Journal 288:6159–6173 (2021).

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Engineering DNA Virus Immune Response 110

Drug Target Review

MAY 16, 2024

The body’s ability to maintain homeostasis in chronic inflammatory and autoimmune diseases is a defining feature of good health, and a goal is largely neglected by modern medicine. This suggests that the field of drug discovery is primed for a paradigm shift in the fundamental strategy of how diseases are treated.

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