FDA Law Blog: Drug Discovery

JULY 17, 2024

Importantly, the Hub is intended to establish a new model within FDA, which leverages cross-Agency expertise in providing guidance and conducting reviews for products for rare disease populations. Early Direction for the Rare Disease Innovation Hub The Hub, which will be co-chaired by Drs. By Sarah Wicks & James E.

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The Pharma Data

NOVEMBER 17, 2020

17, 2020 — From 2009 to 2018, there were increases in diseases-of-despair diagnoses, according to a study published online Nov. Diagnoses related to alcohol misuse, substance misuse, and suicide ideation/behaviors were defined as diseases of despair. TUESDAY, Nov. 9 in BM J Open. Emily Brignone, Ph.D.,

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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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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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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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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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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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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

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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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

NOVEMBER 1, 2023

2 Implication of ATX in a large range of human diseases have been highlighted by both fundamental research and clinical trials. 1-5 Implication of ATX in a large range of human diseases have been highlighted by both fundamental research and clinical trials. 2018 Jun 13;5. 2018 Sep;37(2–3):509–18. Frontiers in Medicine.

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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. We believe we are at a breakthrough moment in Alzheimer’s disease research.

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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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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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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

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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Advarra

JULY 26, 2022

The 2018 Farm Bill. The 2018 Farm Bill removed hemp from the Controlled Substance Act. Importantly, the 2018 Farm Bill preserved FDA authority to regulate products with cannabis or cannabis-derived compounds under the Federal Food, Drug, and Cosmetic (FD&C) Act and Section 351 of the Public Health Service Act.

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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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Codon

NOVEMBER 3, 2024

Physicians working in the early 20th century had little choice but to treat the world’s most rampant infectious disease with methods such as these. But even now, more than a century later, TB remains the deadliest infectious disease on Earth, killing about 1.2 million people every year.

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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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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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New Drug Approvals

OCTOBER 12, 2024

Alzheon licensed ALZ-801 from NeuroChem and is developing it for Alzheimer’s disease. ALZ-801 is an advanced and markedly improved candidate for the treatment of alzheimer’s disease. 2018; 32(9): 849–861. [2]. 2018 Mar;57(3):315-333. 2007 ; Kocis et al., 2016 ; Abushakra et al., Hey JA, et al. Clin Pharmacokinet.

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LifeSciVC

NOVEMBER 17, 2023

This year we start with a deep dive into the biomedical innovation across our industry – “putting the Big back into Big Pharma” with obesity and Alzheimer’s, and the state of play across other disease areas, industry R&D productivity, and the risks facing the sector. As usual, we close with a brief update on Atlas itself.

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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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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.”

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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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KIF1A

MAY 4, 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. By talking to groups with these mutations, we may identify other diseases whose patients have KIF1A variants. She once worked at SACELLS Biotechnology Co.

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Drug Target Review

SEPTEMBER 26, 2023

Human derived induced pluripotent stem cells (hiPSCs) have revolutionised research and are increasingly used for toxicology screening and disease modelling. 2018 Mar;17(3):167–81. 2018 Dec 11;94(10):390–411. 2018;(136):56573. Using stem cell-derived neurons in drug screening for neurological diseases.

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Drug Target Review

NOVEMBER 6, 2023

Advances in disease understanding and combination strategies in haematology We now know that blood cancers are characterised by molecular and phenotypic heterogeneity. Published 2018 Nov 13. 2 It is this complexity that necessitates powerful, targeted combination therapies. Available at: [link] Accessed October 2023. Front Pharmacol.

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New Drug Approvals

SEPTEMBER 23, 2024

Target: DNA/RNA Synthesis Pathway: Cell Cycle/DNA Damage Huntington’s disease (HD) is a progressive, autosomal dominant neurodegenerative disorder of the brain, having symptoms characterized by involuntary movements, cognitive impairment, and mental deterioration. SCHEME PATENT PTC Therapeutics Inc., Audiol Res. 2020-01-02. 2020-01-02.

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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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The Pharma Data

JANUARY 26, 2021

26, 2021 — According to 2018 data, one in five people in the United States probably carries a sexually transmitted infection, the U.S. Centers for Disease Control and Prevention says. On any given day in 2018, nearly 68 million people… Source link. TUESDAY, Jan.

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The Pharma Data

DECEMBER 1, 2020

In 2018, nearly 11% – 14.3 “Individuals with cardiovascular disease have even higher health care costs, including medication costs, so they have to choose what to prioritize,” said Mahajan, a postdoctoral associate at Yale School of Medicine in New Haven, Connecticut. That was compared with 9.1% million – U.S.

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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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The Pharma Data

MAY 5, 2022

Validated Measurable Residual Disease Assays are Critical for the Rapid Development of Future Novel Therapeutics for Patients with Acute Myeloid Leukemia. At Janssen, we’re creating a future where disease is a thing of the past. About Acute Myeloid Leukemia. About the Janssen Pharmaceutical Companies of Johnson & Johnson.

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Drug Target Review

NOVEMBER 3, 2023

The findings may have implications for our understanding of gastrointestinal diseases and endocrine disorders, as well as pancreatic development and diabetes. Further research This discovery may have implications for understanding gastrointestinal diseases and endocrine disorders. 2018 February 8 [2023 November 1];172(4):650-65.

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The Pharma Data

MARCH 16, 2022

The Independent Data Monitoring Committee recommended that the EMPA-KIDNEY trial be stopped early, following a formal interim assessment EMPA-KIDNEY is the largest and broadest dedicated SGLT2 inhibitor trial in chronic kidney disease to date Detailed results are expected to be presented later this year.

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