Eye on FDA

MARCH 7, 2024

Back in December 2023, FDA announced intention in the Federal Register and in a press release to form a new FDA Advisory Committee to be called the Genetic Metabolic Diseases Advisory Committee (GeMDAC).

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Drug Discovery World

SEPTEMBER 4, 2024

The EU OMPD complements the orphan drug designation (ODD) granted by the US FDA in 2020, enhancing the protection and value of CS1 in these major markets. These market exclusivities, alongside our robust patent portfolio, are integral to our commercial strategy for CS1,” said Sten Sörensen, CEO, Cereno Scientific.

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Drug Target Review

MAY 7, 2024

Why are biomarkers particularly critical in the context of rare diseases? Developing novel therapeutic approaches for rare diseases poses inherent challenges due to the limited populations available for testing. How do AI-enhanced approaches accelerate the drug development process for rare diseases?

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DrugBank

JULY 20, 2023

Rare diseases, therefore, present compelling opportunities for Drug Development. Since each disease is present in small groups of patients, this research is also fraught with complex challenges. We're excited to expand our data offering by launching a new Rare Disease add-on to complement our Clinical Trial dataset.

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Drug Discovery World

AUGUST 22, 2024

Talus Bioscience CEO and Co-Founder Alex Federation said: “With our growing team of leading data scientists, we can use tens of millions of data points generated from the MARMOT platform to predict the best places to look for new molecules to block disease-causing transcription factors. million, along with $19.7

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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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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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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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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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Drug Discovery World

MAY 3, 2024

TGCT is a type of tumour that affects the soft tissue lining of joints and tendons and is a highly debilitating disease often impacting large, important joints such as the knee, hip and ankle. It is a chronic disease, which often impacts patients throughout their lives. References Lin F, et. JHEOR, 2022 Cassier , et al.

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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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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

DECEMBER 28, 2020

Centers for Disease Control and Prevention said Saturday. “Today I’m here as a citizen, but most of all as a nurse, to represent my category and all the health workers who choose to believe in science,” Claudia Alivernini, the first person to be vaccinated at the Spallanzani infectious disease hospital in Rome, told the AP.

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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

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

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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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 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

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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Drug Discovery World

NOVEMBER 16, 2023

The Medicines and Healthcare products Regulatory Agency (MHRA) has authorised the world’s first gene therapy for sickle-cell disease (SCD) and transfusion-dependent β-thalassemia (TDT). It is estimated that this includes around 2,000 patients in the UK. Trial results for Casgevy The authorisation is based on positive clinical trial data.

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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 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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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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The Pharma Data

MARCH 24, 2021

billion EUR in 2020 (18.9% Boehringer Ingelheim stepped up its investments in R&D significantly in 2020 in pursuit of innovative medicines and therapies for diseases for which no satisfactory treatments are available. Our achievements in 2020 are the result of their effort.”. Overall R&D investment up 7% to 3.7

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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

OCTOBER 21, 2020

During the third quarter of 2020, the INFOSAN Secretariat has been involved in 37 food safety events involving 78 WHO Member States and five territories of WHO Member States. As of 27 August 2020, 78 cases in the USA have been linked to the outbreak strain of Salmonella Enteritidis; no deaths have been reported. Food safety events.

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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 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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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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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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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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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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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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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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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.

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