This site uses cookies to improve your experience. To help us insure we adhere to various privacy regulations, please select your country/region of residence. If you do not select a country, we will assume you are from the United States. Select your Cookie Settings or view our Privacy Policy and Terms of Use.
Cookie Settings
Cookies and similar technologies are used on this website for proper function of the website, for tracking performance analytics and for marketing purposes. We and some of our third-party providers may use cookie data for various purposes. Please review the cookie settings below and choose your preference.
Used for the proper function of the website
Used for monitoring website traffic and interactions
Cookie Settings
Cookies and similar technologies are used on this website for proper function of the website, for tracking performance analytics and for marketing purposes. We and some of our third-party providers may use cookie data for various purposes. Please review the cookie settings below and choose your preference.
Strictly Necessary: Used for the proper function of the website
Performance/Analytics: Used for monitoring website traffic and interactions
By Lucy Mutz November 4, 2024 Credit: Juliana Sohn Kasper Lage is managing director of the Novo Nordisk Foundation Center for Genomic Mechanisms of Disease. The overall goal is to translate genetic findings into biological mechanisms underlying disease that could be targeted with new medicines.
The search for effective treatments for neurodegenerative diseases like Parkinson’s disease has long been hindered by the brain’s complexity and the absence of adequate models for drug discovery. “For diseases like Parkinson’s, it’s more than sufficient,” he explains.
Despite recent advances in gene therapy for sickle cell disease (SCD) , automated red blood cell exchange (aRBCX) remains a cornerstone therapy that plays a vital yet underutilised role in managing complications and enhancing quality of life for millions living with this devastating condition worldwide.
By Allessandra DiCorato January 14, 2025 Credit: Maria Nemchuk, Broad Communications Eric Minikel and Sonia Vallabh run a lab with a singular focus: preventing and treating prion disease within their lifetime. Its a huge privilege to have the opportunity to point these powerful new tools at our disease.
Introduction Therapeutic antibodies have proven to be indispensable medicines for addressing the most debilitating diseases. Agonist antibodies in immunological diseases Agonist antibodies targeting immune checkpoint pathways are increasingly recognised and investigated for their potential in immunological diseases.
The extended agreement will support joint research efforts targeting critical therapeutic areas, including oncology, cardiovascular and renal diseases, neurology, rare diseases, and immunology. Oncology remains a core priority, reflecting the rising global burden of cancer and the need for more effective, targeted therapies.
Consequently, these drugs have limited ability to keep patients disease-free and limited impact on patient survival. These therapeutics share some characteristic measures of clinical efficacy, including high initial overall response rates but a low rate of complete responses and limited duration of response. Lancet Haem 11: e693-e707.
Eligible participants had experienced disease progression on one or two prior lines of endocrine therapy , including one line with a CDK4/6 inhibitor , and could have received up to one prior line of chemotherapy in the advanced or metastatic setting. [2] 1] [4] It is taken by mouth. [1] 3] [7] PATENTS Cruskie MP, et al.
15 February 2018). 3] Primary endocrine resistance was defined as relapse while on the first two years of adjuvant endocrine therapy (ET) and secondary endocrine resistance was defined as relapse while on adjuvant ET after at least two years or relapse within twelve months of completing adjuvant ET. [3] doi : 10.1038/ni0403-313.
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.
Founding Vision: Harnessing RNA Editing for Transformative Medicine Korro Bio’s story began in 2018, co-founded by a remarkable group: Jean-François Formela, M.D. was based on experience building on the understanding that a single protein variant, could have markedly different outcomes for patients with chronic kidney disease.
Its sodium salt is used for the treatment of generalised myasthenia gravis (a disease that leads to muscle weakness and tiredness) in adults whose immune system produces antibodies against acetylcholine receptors. April 2018). World Health Organization (2018). 4 December 2023. Archived from the original on 11 December 2023.
This update is based on robust findings from the STRIDE trial , a landmark study examining the drug’s efficacy in peripheral artery disease (PAD) —a common, debilitating, and often overlooked complication in individuals with type 2 diabetes (T2D). FLOW Trial: The study focused on chronic kidney disease (CKD) in type 2 diabetes patients.
On a conference call held Wednesday afternoon, Sarepta CEO Doug Ingram said that "failure to adapt" to recent setbacks "would risk our long-term viability as an organization and decrease the opportunity to bring the greatest benefit to the greatest number of patients living with rare disease." Track how they’re performing.
2 Drug use contributes to the spread of HIV and other infectious diseases like hepatitis B and C in multiple ways, including the sharing of drug injection equipment. According to a 2018 modeling study, at least 127 infections could have been prevented had an SSP opened two years sooner than it did. 2018 Oct;5(10):e569-e577.
HSV in immunocompromised patients Although HSV infection is very common in the general population, it rarely causes serious disease and is effectively contained by the immune system. Infectious Diseases Community of Practice (2013). Infectious Diseases Community of Practice (2009). 2018 Oct 23;9:2483. 2018 Jan;149:1-6.
A number of studies have shown that blocking the CSF-1R signaling pathway could effectively modulate and change macrophage functions, and potentially treat many macrophage-dependent human diseases. [1] 2018-11-29. [2]. Cancer Research, 2018, 78(13_Supplement): LB-288-LB-288. [3]. 6] The U.S. mmol) in methanol (10 mL).
Last year, an American Heart Association presidential advisory for the first time formally identified the strong connections between cardiovascular disease (CVD), kidney disease, Type 2 diabetes and obesity as reason to define cardiovascular-kidney-metabolic (CKM) syndrome.
Permission granted by Incyte Dive Brief: Incyte has named veteran pharmaceutical executive Bill Meury as its new CEO, replacing longtime head Hervé Hoppenot, who led the cancer and blood disease drugmaker for the past 11 years. However, it flamed out in testing in 2018 and Incyte was forced to pivot research toward other candidates.
Daily exposure to certain chemicals used to make plastic household items could be linked to more than 356,000 global deaths from heart disease in 2018 alone, a new analysis of population surveys shows.
13, 2024 -- Obesity, alcohol use and other factors are driving up rates of fatty liver disease among American adults, new research warns.By 2018, federal data showed that 42% of adults had some form of fatty liver disease -- higher. WEDNESDAY, Nov.
FRIDAY, June 13, 2025 — Alcohol-related liver disease deaths are increasing — and they’re rising faster in some groups, including women, young adults and Indigenous people, new research shows.Between 2018 and 2022, deaths from alcohol-associated liv.
Having already been on a winding journey, Enjaymo, which Sanofi acquired through a 2018 buyout of Bioverativ, is now headed to Recordati as part of a deal announced Friday.
A medicine the pharma acquired in a $430 million buyout of Visterra in 2018 succeeded in a Phase 3 trial in IgA nephropathy, a crowded corner of drug research.
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.,
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.
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.
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.
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 […]
An antibody drug, Beyfortus , that Sanofi developed with partner AstraZeneca has become a blockbuster product for preventing RSV disease in infants. This “ molecular clamp ” platform is designed to elicit strong immune responses against respiratory diseases, while permitting simpler and faster production and distribution.
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.
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.
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.
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.
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.
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.
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.
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.,
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.
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.
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. .
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.
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.”
We organize all of the trending information in your field so you don't have to. Join 15,000+ users and stay up to date on the latest articles your peers are reading.
You know about us, now we want to get to know you!
Let's personalize your content
Let's get even more personalized
We recognize your account from another site in our network, please click 'Send Email' below to continue with verifying your account and setting a password.
Let's personalize your content