Drug Target Review

OCTOBER 11, 2023

Almost every one of us will know someone with a neurodegenerative disease such as Alzheimer’s or Parkinson’s. It is less common to know someone with a rare genetic disease; but while individually rare, collectively they have an enormous impact. This will, in turn, slow the progression of, or even halt, the disease.

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SCIENMAG: Medicine & Health

JUNE 16, 2023

CHICAGO—The percent of metabolic associated fatty liver disease (MAFLD), the leading global cause of liver disease, is increasing in U.S. Mexican Americans consistently had the highest percentage of MAFLD, especially in 2018, although the prevalence of increase […]

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Drug Target Review

AUGUST 7, 2023

This finding holds the promise of potentially slowing down the aging clock, allowing individuals to lead longer, healthier lives, while also providing potential avenues to prevent age-related conditions, such as heart disease and neurodegenerative disorders. Oxford, United Kingdom: Oxford University Press; 2018. References Lane N.

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The Pharma Data

NOVEMBER 19, 2020

2010 to 2018 Saw Decrease in Rate of Death for People With HIV. 19, 2020 — From 2010 to 2018, there was a 36.6 Centers for Disease Control and Prevention Morbidity and Mortality Weekly Report. percent overall during 2010 to 2018 (from 19.4 Professional. THURSDAY, Nov. 20 issue of the U.S. Bosh, Ph.D., to 8.5).

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Broad Institute

JULY 6, 2023

The accuracy of these scores has improved for some diseases and groups of people, but they continue to fall short for those of non-European ancestry, mainly because the genetic datasets used to calculate these scores have largely come from people of European ancestry.

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FDA Law Blog: Drug Discovery

DECEMBER 13, 2023

Sasinowski — On December 12, 2023, FDA announced the creation of a new advisory committee specifically for treatments for genetic metabolic diseases, the Genetic Metabolic Diseases Advisory Committee, or “GeMDAC.” There are hundreds of known genetic metabolic diseases, most of which are rare and carry significant morbidity.

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Drug Target Review

JULY 13, 2023

Dewpoint Therapeutics, a platform drug discovery company founded in 2018, has been at the forefront of this exciting field. Dewpoint’s breakthrough was the realisation that certain condensates are associated with human diseases. Klein notes the importance of Dewpoint’s disease-agnostic approach.

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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.

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Conversations in Drug Development Trends

FEBRUARY 28, 2023

95% of rare diseases don’t have a treatment approved and won’t anytime soon. Uplifting Athletes As a 501(c)(3) nonprofit, UA harnesses the power of sport and partnerships with patient advocacy groups to fund the researchers developing datasets for rare diseases that have never been studied or understood before.

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Broad Institute

SEPTEMBER 15, 2023

We apply STACI to analyze the spatio-temporal progression of Alzheimer’s disease and identify the associated nuclear morphometric and coupled gene expression features.

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Disease 40

BioPharma Drive: Drug Pricing

MARCH 17, 2024

The biotech, formed as a successor to a company Roche acquired in 2018, has two drugs in clinical testing for neurological and immune diseases.

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Drug Discovery World

MARCH 12, 2024

Three of these are key to the advancement of gene therapies, cancer research and Parkinson’s treatments.

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Drug Discovery World

DECEMBER 5, 2022

A new partnership between industry and three rare disease foundations has formed with the goal of accelerating the development of precision drugs to treat rare neurodevelopmental disorders. . Understanding rare diseases remains a significant challenge. Understanding rare diseases remains a significant challenge.

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Drug Discovery World

DECEMBER 18, 2023

Casgevy The committee recommended granting a conditional marketing authorisation for Casgevy (exagamglogene autotemcel), an advanced therapy medicinal product (ATMP) for the treatment of transfusion dependent beta-thalassemia and severe sickle cell disease.

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Drug Discovery World

SEPTEMBER 1, 2023

metaLead, which is developing therapies to treat metal-related diseases, currently focused on Wilson disease, lead poisoning, and neurodegeneration with brain iron accumulation. Amalus Therapeutics, which is reshaping the lives of people affected by cancer and fibrotic diseases.

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The Pharma Data

JANUARY 27, 2021

27, 2021 — Heart disease remains the leading cause of death worldwide, with 18.6 million deaths attributed to cardiovascular disease (CVD) globally in 2019, according to a report from the American Heart Association published online Jan. WEDNESDAY, Jan. 27 in Circulation. Virani, M.D., Virani, M.D., percent from 2010.

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The Pharma Data

DECEMBER 17, 2020

17, 2020 — Lower socioeconomic status (LSS) is associated with worse outcomes for inflammatory bowel disease (IBD), according to a study published in the December issue of The American Journal of Gastroenterology. For those with Crohn disease, the impact of LSS was greater than for those with ulcerative colitis. THURSDAY, Dec.

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Drug Discovery World

APRIL 11, 2023

2018; 10 (1): 34‐45 Duchesne M, et al. Elevated Expression of IL-33 and TSLP in the Airways of Human Asthmatics In Vivo: A Potential Biomarker of Severe Refractory Disease. 2018; 200: 2253–2262. There is also a significant healthcare burden, with these patients accounting for approximately 50% of NHS asthma-related costs.

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The Pharma Data

NOVEMBER 14, 2020

14, 2020 — Vaping significantly increases the risk of lung disease, according to a new study that adds to growing evidence that e-cigarettes are dangerous. SATURDAY, Nov. Researchers from Boston University analyzed data gathered from more than 21,600 U.S.

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The Pharma Data

DECEMBER 4, 2020

Alzheimer Disease Tied to Adverse Financial Events. 4, 2020 — Alzheimer disease and related dementias (ADRDs) are associated with adverse financial events years prior to clinical diagnosis, according to a study published online Nov. Professional. FRIDAY, Dec. 30 in JAMA Internal Medicine. Lauren Hersch Nicholas, Ph.D.,

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Disease International Treatment Research 52

PLOS: DNA Science

JUNE 1, 2023

This DNA Science post from 2018 traces the history of the efforts. Peter Marinkovich, MD, director of the Blistering Disease Clinic at Stanford Health Care, calls DEB a devastating disease. A dominant form – DDEB – is less severe, blistering the hands, feet, knees, and elbows. Primary investigator M.

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Drug Discovery World

JULY 14, 2022

The same formulation of tecovirimat was approved by the US Food and Drug Administration (FDA) in 2018 and by Health Canada in late 2021 under the brand name TPOXX?for As we have learned in the ongoing COVID-19 pandemic, building robust stockpiles in response to infectious disease outbreaks is of vital importance worldwide.

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Drug Discovery World

SEPTEMBER 15, 2022

CRC is the second most common cause of cancer mortality, accounting for 881,000 deaths globally in 2018, and patients with metastatic disease have a five-year survival rate of just 11%. The Phase III clinical trial studied participants with refractory metastatic colorectal cancer (mCRC) following two chemotherapy regimens.

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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.

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SCIENMAG: Medicine & Health

JUNE 13, 2023

People who adhere to global Cancer Prevention Recommendations are putting themselves at lower risk of developing the disease, new research confirms. Experts at Newcastle University have reviewed evidence of following the 2018 World Cancer Research Fund (WCRF) and American Institute for Cancer Research (AICR) lifestyle-based recommendations.

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Drug Discovery World

MARCH 6, 2023

Since its inception in 2018, BII has supported 80 start-ups and projects with €65 million alongside the venture capital, industry and business expertise it provides to help them accelerate to the next level.

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Drug Discovery World

JANUARY 23, 2023

Following the release of Clarivate’s report ‘RNA Technology Companies to Watch’, DDW’s Diana Spencer speaks to Strand Therapeutics’ Jacob Becraft and HAYA Therapeutics’ Samir Ounzain to find out how this technology could revolutionise disease treatment. DS: How will your work ultimately impact disease treatment?

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Agency IQ

MAY 17, 2024

The Creating Hope Reauthorization Act would reauthorize the FDA’s Rare Pediatric Disease Priority Review Voucher program. The markup hearing comes three months after the E&C Committee last held a hearing on 18 bills related to rare diseases, including all four of the above-mentioned bills. DORIS MATSUI (D-Calif.)

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The Pharma Data

AUGUST 24, 2020

AstraZeneca and RenalytixAI have agreed a deal to develop joint precision medicine strategies for cardiovascular, renal and metabolic diseases. The companies will first target chronic kidney disease and use RenaltytixAI’s diagnostic platform, called KidneyIntelX, to examine how to improve patient outcomes.

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Drug Discovery World

APRIL 30, 2024

Whilst research into mRNA has been ongoing for decade, interest has sparked in recent years and research has begun to expand into areas of vaccinology, infectious diseases and other indications. For instance, consider how at the end of 2018, the mRNA therapies market was valued at $3.43

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The Pharma Data

OCTOBER 26, 2020

Food and Drug Administration (FDA) has approved EYSUVIS for the short-term treatment of dry eye disease. . adults have been diagnosed with dry eye disease, a chronic, episodic, multifactorial disease. It also increases your risk for inflamed cornea, cornea eye disease, corneal ulcers and injuries, and eye infections. .

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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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Drug Discovery World

MARCH 17, 2023

Since 2018, GARDP and the University of Liverpool have been working together to advance new treatment options for new-borns with sepsis. The University of Liverpool is a leader in infectious disease research and a key partner in GARDP’s work on children’s antibiotics,” said Seamus O’Brien, R&D Director at GARDP.

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The Pharma Data

OCTOBER 28, 2020

Johnson & Johnson has submitted a supplemental New Drug Application to the FDA seeking approval for Xarelto (rivaroxaban) plus aspirin for patients suffering from peripheral artery disease (PAD). The application is supported by data showing that Xarelto plus aspirin reduced the risk of major cardiovascular and limb adverse events.

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Agency IQ

APRIL 19, 2024

FDA advisors endorse minimal residual disease (MRD) as accelerated approval endpoint for multiple myeloma Last week, FDA’s Oncologic Drugs Advisory Committee (ODAC) voted unanimously in favor of using minimal residual disease (MRD) as an accelerated approval endpoint for multiple myeloma.

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SCIENMAG: Medicine & Health

SEPTEMBER 12, 2023

In the summer of 2018, graduate student Hunter Dean from the Medical Scientist Training Program came to Yuhua Song, Ph.D., He wanted to pursue his doctoral research at the University of Alabama at Birmingham in Alzheimer’s disease — under the joint mentorship of Song, who had never worked […] BIRMINGHAM, Ala. –

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The Pharma Data

DECEMBER 3, 2020

3, 2020 (American Heart Association News) — Your chances of dying from heart disease or stroke are higher if you live in a county considered socially vulnerable due to factors such as poverty, crowded housing and poor access to transportation, new research shows. THURSDAY, Dec. More than 1 in 3 U.S.

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