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

FEBRUARY 14, 2024

Researchers from the Michigan State University have created an advanced human heart organoid system that models the characteristics of pregestational diabetes-induced congenital heart disease found in mice and humans. The most common type of congenital defect in humans is congenital heart disease.

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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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The Pharma Data

OCTOBER 21, 2020

22, 2020 (GLOBE NEWSWIRE) — Agios Pharmaceuticals, Inc. NASDAQ: AGIO), a leader in the field of cellular metabolism to treat cancer and rare genetic diseases, today announced that the company will host a conference call and live webcast on Thursday, November 5, 2020 at 8:00 a.m. CAMBRIDGE, Mass.,

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International Disease Therapies Pharmaceuticals 52

The Pharma Data

DECEMBER 2, 2020

Centers for Disease Control and Prevention. Centers for Disease Control and Prevention. © 2020 HealthDay. Posted: December 2020. Below are newsworthy items compiled by the Healthday staff: CDC To Shorten Recommended Quarantine Period. The antibodies suggest exposure to the new coronavirus or one very similar to it.

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Virus Disease Research 52

The Pharma Data

NOVEMBER 13, 2020

Centers for Disease Control and Prevention analysis finds. “While health systems are strained by the COVID-19 pandemic, we must not allow our fight against one deadly disease to come at the expense of our fight against another. © 2020 HealthDay. Posted: November 2020. All rights reserved. Source link.

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The Pharma Data

NOVEMBER 9, 2020

An experimental Alzheimer’s disease treatment should not be approved by the U.S. The drug, developed by the pharmaceutical company Biogen and its Japanese partner Eisai, is administered through intravenous infusion for early Alzheimer’s disease. © 2020 HealthDay. Posted: November 2020. All rights reserved.

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Pharmaceutical Companies Treatment FDA Disease 52

The Pharma Data

OCTOBER 14, 2020

15, 2020 (GLOBE NEWSWIRE) — Eloxx Pharmaceuticals, Inc. Conference Call Information : Date : Thursday, November 5, 2020. These premature stop codons have been identified in over 1,800 rare and ultra-rare diseases. These premature stop codons have been identified in over 1,800 rare and ultra-rare diseases.

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Science Daily: Pharmacology News

SEPTEMBER 19, 2024

The study is based on a new analysis of metatranscriptomic data released by the Chinese Center for Disease Control and Prevention (CDC). The data come from more than 800 samples collected in and around the Huanan Seafood Wholesale market beginning on January 1, 2020, and from viral genomes reported from early COVID-19 patients.

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The Pharma Data

DECEMBER 16, 2020

The objective of the 10-person WHO team is to gather information to better prepare for possible future outbreaks, according to team member Fabian Leendertz, a German biologist who specializes in emerging diseases. © 2020 HealthDay. Posted: December 2020. In total, the mission is expected to last four to five weeks.

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Virus Disease 52

The Pharma Data

DECEMBER 21, 2020

While they expect vaccines will be effective against the new variant, they should know for sure within a few days, according to Dr. Nelson Michael, director of the Center for Infectious Diseases Research at the Walter Reed Army Institute of Research. Centers for Disease Control and Prevention. © 2020 HealthDay. Source link.

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The Pharma Data

NOVEMBER 20, 2020

Centers for Disease Control and Prevention shows. © 2020 HealthDay. Posted: November 2020. Adult Vaping Rate Rises, Smoking Rate Remains Same. The rate of smoking among American adults seems to have leveled off, while vaping appears to be on the rise, a new survey from the U.S. We are sounding the alarm,” Gov.

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Virus Disease 52

The Pharma Data

NOVEMBER 2, 2020

Centers for Disease Control and Prevention says. © 2020 HealthDay. Posted: November 2020. Here’s a collection of newsworthy items compiled by Healthday News staff: CDC Issues Pandemic Safety Guidelines for Cruise Ships. He won an Academy Award as best supporting actor for his role in the 1987 movie “The Untouchables.”

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Doctors Disease 52

The Pharma Data

DECEMBER 30, 2020

The year 2020 will certainly go down in history as the year of COVID-19. Gilead Sciences Makes Multiple Bets : When Gilead Sciences wasn’t working on remdesivir in COVID-19, the company seemed to be constantly flexing its M&A muscle over the course of 2020. It began in May with the $4.9 But the U.K.-based

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The Pharma Data

OCTOBER 20, 2020

21, 2020 /PRNewswire/ — Ionis Pharmaceuticals, Inc. Eastern Time to discuss its third quarter 2020 financial results and report on pipeline and business progress. Webcast scheduled for Wednesday, November 4 at 11:30 a.m. Eastern Time. . CARLSBAD, Calif. , Interested parties may access the webcast at www.ionispharma.com.

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RNA Pharmaceuticals Disease Treatment 52

The Pharma Data

NOVEMBER 30, 2020

30, 2020 /PRNewswire/ — Regeneron Pharmaceuticals, Inc. NASDAQ: REGN) will host a webcast on Monday, December 7, 2020 at 4:30pm EST. Management will discuss data presented at the American Society of Hematology (ASH) 2020 Annual Meeting as well as provide updates on the Company’s broader oncology and hematology portfolio.

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FDA Approval Laboratories Disease Pharmaceuticals 52

The Pharma Data

NOVEMBER 8, 2020

Verona Pharma is a clinical-stage biopharmaceutical company focused on developing and commercializing innovative therapies for the treatment of respiratory diseases with significant unmet medical needs. LONDON and RALEIGH, N.C., For more information, please visit www.veronapharma.com. Forward-Looking Statements.

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The Pharma Data

JANUARY 12, 2021

(NASDAQ: NARI) (“Inari”) a commercial-stage medical device company focused on developing products to treat and transform the lives of patients suffering from venous diseases, today reported preliminary unaudited fourth quarter 2020 revenue. million to $48.9 million to $48.9 About Inari Medical, Inc. Inari Medical, Inc.

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The Pharma Data

NOVEMBER 18, 2020

Antimicrobial resistance occurs when microorganisms resist the effects of medications, making infections harder to treat and increasing the risk of disease spread, severe illness and death. Map of countries implementing national HIV drug resistance surveys, 2004–2020. . Key definitions. Combatting antimicrobial resistance is a global.

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The Pharma Data

SEPTEMBER 28, 2020

Although Chagas disease was discovered more than a century ago, it remains a major public health challenge. Chagas disease affects approximately six million people, mainly in Latin America. Like other neglected tropical diseases, Chagas disease often affects poor and marginalized communities. Chagas Disease.

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Disease Treatment 52

The Pharma Data

DECEMBER 9, 2020

10, 2020 — Heart disease is the leading cause of death worldwide — accounting for one-third of deaths in 2019 — and the death toll continues to rise, a new paper says. China had the highest number of heart disease deaths last year, followed by India, Russia, the United States and Indonesia. © 2020 HealthDay.

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Disease 52

The Pharma Data

OCTOBER 27, 2020

Infectious Disease Least Severe in School-Aged Children. 27, 2020 — School-aged children seem to have the least severe disease for different infectious diseases, according to a study published online Oct. © 2020 HealthDay. . © 2020 HealthDay. Posted: October 2020. Professional. TUESDAY, Oct.

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The Pharma Data

JANUARY 12, 2021

12, 2021 — During the initial phase of the COVID-19 pandemic, there was an increase in deaths caused by ischemic heart disease and hypertensive diseases in the United States, as well as a reduction in cardiovascular diagnostic testing across the world, according to two studies published in the Jan. TUESDAY, Jan. Einstein, M.D.,

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Disease Hospitals Pharmaceuticals Research 52

The Pharma Data

JANUARY 15, 2021

Breast, Cervical, CRC Screening Below Healthy People 2020 Targets. 15, 2021 — Receipt of screening tests for breast, cervical, and colorectal cancers was below national Heathy People 2020 targets in 2018, according to research published in the Jan. Centers for Disease Control and Prevention Morbidity and Mortality Weekly Report.

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The Premier Consulting Blog

JUNE 18, 2023

One exciting application of these technologies is the use of in silico trials in the development of novel therapies for rare diseases. However, with rare diseases there may be no available treatments that could serve as SoC or active control in a clinical trial and assigning patients to placebo may be unethical.

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Drug Development Disease Clinical Trials Drugs 52

The Pharma Data

DECEMBER 31, 2020

Monthly News Roundup – December 2020. In studies, the vaccines were found to be 94% to 95% effective in preventing COVID-19 disease. The partial piece of the spike protein cannot cause COVID-19 disease. Posted: December 2020. Moderna and Pfizer’s COVID-19 Vaccines Roll Out in Early Phase.

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FDA Approval Vaccine FDA Treatment 52

The Pharma Data

OCTOBER 15, 2020

Vertex Pharmaceuticals has decided to give up on its experimental VX-814, a small molecule drug for the rare genetic disease Alpha-1 antitrypsin deficiency (AATD), canning the drug’s development after seeing lackluster results from an early phase 2 trial. James Miessler.

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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. © 2020 HealthDay. Posted: November 2020. SATURDAY, Nov. Researchers from Boston University analyzed data gathered from more than 21,600 U.S. All rights reserved.

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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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The Pharma Data

NOVEMBER 4, 2020

4, 2020 — Overall, 1.4 percent of patients with COVID-19 have acute cerebrovascular disease, with acute ischemic stroke being the most common manifestation, according to a review published online Oct. The researchers found that acute cerebrovascular disease occurred in 1.4 © 2020 HealthDay. Posted: November 2020.

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Disease Hospitals International Research 52

The Pharma Data

DECEMBER 15, 2020

15, 2020 — Patients diagnosed with depression in youth have increased risks for many somatic diseases and mortality, according to a study published online Dec. The absolute risk difference of a specific disease within 12 years of first depression diagnosis varied from ?0.2 © 2020 HealthDay. Posted: December 2020.

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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. 2020-01-02. SCHEME PATENT PTC Therapeutics Inc., Audiol Res. J Pers Med.

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Small Molecule RNA DNA Disease 57

The Pharma Data

NOVEMBER 13, 2020

Mental Health ED Visits in Children Up Mid-March to October 2020. 13, 2020 — From mid-March to October 2020, there was an increase in the proportion of mental health-related emergency department visits among children and adolescents, according to research published in the Nov. 17, 2020, compared with 2019.

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Disease Research 52

The Pharma Data

JANUARY 21, 2021

Percent of Kindergarteners Not Up to Date With MMR in 2019-2020. percent of kindergarteners were not up to date with measles, mumps, and rubella (MMR) vaccination and did not have an exemption in the 2019 to 2020 school year, according to research published in the Jan. Professional. THURSDAY, Jan. 21, 2021 — Overall, 2.3

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Vaccine Disease Research 52

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. © 2020 HealthDay. Posted: December 2020. Professional. FRIDAY, Dec.

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Disease International Treatment Research 52

The Pharma Data

SEPTEMBER 16, 2020

16, 2020 /PRNewswire/ — Amgen (NASDAQ:AMGN) will host two webcast calls for the investment community in conjunction with the European Society for Medical Oncology (ESMO) 2020 Virtual Congress. 20, 2020, at 11:00 a.m. THOUSAND OAKS, Calif. On Sunday, Sept. PDT, David M. Reese , M.D.,

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Clinical Development Therapies Disease Research 52

The Pharma Data

OCTOBER 26, 2020

27, 2020 /PRNewswire/ — Synlogic, Inc. Nasdaq: SYBX), a clinical stage company bringing the transformative potential of synthetic biology to medicine, today announced the Company will release its third quarter 2020 financial results before the market opens on Thursday, November 5, 2020. CAMBRIDGE, Mass. ,

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