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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.
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.
The latest technologies are empowering researchers with faster, more efficient and more accessible genomic tools, leading to richer data generation and accelerating progress in areas like cancer genomics, rare disease research and infectious disease surveillance. 2021 [cited 2025 Jun 11]. 2021 [cited 2025 Jun 11].
The organization has honored Liu for the development of the gene editing platforms base editing and prime editing, which can correct the vast majority of known disease-causing genetic variations and have already been used in at least 15 clinical trials, with life-saving results.
Drug development faces significant challenges: long timelines, high costs , complex processes and low probabilities of success (PoS), exacerbated by the shift towards more complex molecules, biologics and cell and gene therapies, hindering patient access to vital treatments. Nature, 596, 590-596 (2021) [link] Liu R, Rizzo S, Waliany S, et al.
Despite challenges as a woman in the field, Dr Hingorani built a career focused on advancing treatments for pediatric and adult cancers. Despite the progress we are making in curing children with cancer, there remains an unmet need for new treatments that are more effective and safer.
Multiomics MSI studies provide crucial insights into drug pharmacology and toxicity, supporting the development of innovative therapies including chimeric antigen receptor (CAR) T-cell treatments, oligonucleotide therapeutics, antibody drug conjugates (ADCs) and ribonucleic acid (RNA) therapeutics. 2021, 56 (8), e4717. Mass Spectrom.
Patients diagnosed with idiopathic pulmonary fibrosis (IPF) — a progressive, rare lung disease marked by scarring and worsening lung function — often face a poor prognosis. While two approved treatments, pirfenidone and nintedanib, slow disease progression, there currently is no treatment that reverses the effects or offers a cure for IPF.
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.
link] Ibuzatrelvir (1) was recently disclosed and patented by Pfizer for the treatment of severe acute respiratory syndrome coronavirus 2 (SARS-CoV-2). “A Second-Generation Oral SARS-CoV-2 Main Protease Inhibitor Clinical Candidate for the Treatment of COVID-19” Journal of Medicinal Chemistry. 2021-12-16. [2].
Consequently, these drugs have limited ability to keep patients disease-free and limited impact on patient survival. Other mechanisms that cancer cells use to escape from TCE treatment are less expected. This provides ample flexibility to optimize the treatment modality to attack specific cancers. Oncoimmunology 11: 2111904.
8] Therefore, it may serve as a new addition to combination therapy with conventional cancer treatment, such as chemotherapy. Combining inavolisib with palbociclib and fulvestrant might improve treatment of breast cancer. [14] Vanhaesebroeck B, Perry MW, Brown JR, Andr F, Okkenhaug K (October 2021). Hz, 1H), 6.41 (dd, J=8.8,
Nevadistinel can be used to inhibit cognitive impairment associated with neurodegenerative diseases, such as mild cognitive impairment, mild Alzheimer’s disease, Parkinson’s disease, Lewy body disease. UNII-52TU5MZG22, X062KF5ZV3 (αS,4R)-α-[(1R)-1-Hydroxyethyl]-5-(2-methyl-1-oxopropyl)-1-oxo-2,5-diazaspiro[3.4]octane-2-acetamide
Importantly, this will enable them to produce mRNA vaccines during periods between health crises, retaining trained personnel, technical expertise, and production capacity while retaining the ability to respond quickly in the event of future disease outbreaks. mRNA Technology Transfer Programme Moves Into Phase 2.0,
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. Retrieved 24 June 2021. 6] [9] [10] It is a complement inhibitor that is injected subcutaneously (under the skin).
Medical uses Taletrectinib is indicated for the treatment of adults with locally advanced or metastatic ROS1-positive non-small cell lung cancer. [1] 3] Participants may have received prior chemotherapy for advanced disease. [3] 3] Participants may have received prior chemotherapy for advanced disease. [3] mol, 1 equiv.),
“Second Generation Oral Mpro Inhibitor for COVID-19 Treatment Proceeds in Phase 3 Study” Precision Vaccinations. Jump up to: a b “Coronavirus disease 2019 (COVID-19) emerging treatments” BMJ Best Practice US. Berdowska I, Matusiewicz M (October 2021). mol) and N-hydroxy succinimide (157.1
1] [11] In December 2023, the US Food and Drug Administration (FDA) expanded the indication for pirtobrutinib to include the treatment of adults with chronic lymphocytic leukemia or small lymphocytic leukemia. [7] 7] [12] In the European Union, pirtobrutinib is indicated for the treatment of mantle cell lymphoma. [2] 27 January 2023.
Published June 26, 2025 Gwendolyn Wu Senior reporter post share post print email license B cells, like those pictured above, malfunction in autoimmune disease. Progress developing a treatment for leukemia led the company in 2021 to price a $177 million initial public offering. Data from that trial is expected in 2027. “I
Formula C 27 H 29 Cl 2 FN 2 OS Crinecerfont , sold under the brand name Crenessity , is a medication used for the treatment of congenital adrenal hyperplasia. [1] 4] A medication used to reduce the amount of steroid replacement required in patients with a genetic disease that causes, amongst other symptoms, a steroid deficiency. .
Contrary to worries that syringe services programs (SSPs) will encourage or promote drug use, evidence shows that they more often do the opposite, linking people with addiction to effective treatment and even helping prevent overdose deaths. 6 SSPs also may distribute fentanyl test strips. 6 SSPs also may distribute fentanyl test strips.
Amodei also imagines the ways AI could accelerate biological research and yield miraculous cures in the 21st century; everything from the prevention and treatment of nearly all infectious and inherited diseases to the elimination of most cancers. This essay focuses on how we might do both, specifically for the cell.
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.
OGPhoto via Getty Images Dive Brief: Organon’s experimental treatment for endometriosis failed to improve pelvic pain for women in a Phase 2 study, leading the company to give up on the drug. Executives had been especially excited about OG-6219 because it was a non-hormonal treatment with a new mechanism of action. Can they keep it?
has agreed to pay $10 billion to buy London-based Verona Pharma, scooping up a potential multibillion-dollar new drug to treat chronic obstructive pulmonary disease in a deal announced Wednesday. The Food and Drug Administration approved Verona’s Ohtuvayre in June 2024 for maintenance treatment of COPD in adults. Alamy Merck & Co.
Klarman joined the Broad Institute Board of Directors when it was formed in 2009 and has worked closely with Schmidt and other fellow members to steward the institution in its mission of improving human health by advancing the understanding and treatment of disease.&
As healthcare professionals, it’s our responsibility to educate patients about generic drugs and empower them to make informed decisions about their treatment options. ” FDA.gov, 2021. ” AccessibleMeds.org, 2021. Food and Drug Administration. “Generic Drugs: Questions & Answers.” Shrank, W.H.,
The EIC Accelerator Programme is part of the broader Horizon Europe 2021–2027 Research and Innovation framework, which provides non-dilutive and equity funding to breakthrough, high-risk innovations with global market potential. CroíValve’s DUO™ System presents a novel approach to TR treatment. CroíValve Awarded €12.5M
A Legacy of Innovation and Partnership RTS,S, which became the first malaria vaccine recommended by the World Health Organization (WHO) in 2021, represents a culmination of decades of scientific research and public-private collaboration. No single intervention is sufficient on its own. Source link
3] It was discovered for the treatment of hepatitis C [4] and has since been investigated for use against other viral diseases such as AIDS and COVID-19 , [2] [5] for which it was granted conditional approval in China. [6] It was approved in China in 2021 as a first-in-class treatment for human immunodeficiency virus (HIV).
(Nasdaq: BIIB) today announced it will share multiple oral and poster presentations from its Alzheimer’s disease clinical development portfolio at the Alzheimer’s Association International Conference (AAIC), which will be held in Denver, Colorado and online July 26-30, 2021.
These therapies have broadened treatment options for patients to expand beyond the more traditional small molecule drug alternatives. ADCs have the potential to redress the poor balance between safety and efficacy seen with traditional cancer treatment options.
Biogen (Nasdaq: BIIB) today announced it will host webcasts of its pre-recorded presentations and live discussions related to its Alzheimer’s disease investigational therapy, aducanumab, at the upcoming AD/PDTM 2021 Virtual Conference. AD/PD Webcast Schedule Details: Wednesday, March 10, 2021, 6:45 a.m.
Nasdaq:BIIB) today announced it will report second quarter 2021 financial results Thursday, July 22, 2021, before the financial markets open. Biogen discovers, develops, and delivers worldwide innovative therapies for people living with serious neurological and neurodegenerative diseases as well as related therapeutic adjacencies.
today announced it will report first quarter 2021 financial results Thursday, April 22, 2021, before the financial markets open. Biogen discovers, develops, and delivers worldwide innovative therapies for people living with serious neurological and neurodegenerative diseases as well as related therapeutic adjacencies.
The findings supported the possibility put forward by Cotton et al (2016) concerning the chance of repurposing these drugs for adulticidal treatment. The available treatment for that is minor surgery -to remove the nodules. Need for adulticidal drugs More than 99 percent of countries worldwide has been affected by this disease.
Advancing reduction of drug use as an endpoint in addiction treatment trials astewart Thu, 03/06/2025 - 09:59 Nora's Blog March 18, 2025 Image Getty Images/ SolStock This blog was also published in the American Society of Addiction Medicine (ASAM) Weekly on March 18, 2025.&
to $6.14; Raises 2021 Adjusted Diluted EPS Guidance Range from $12.37 Based upon the momentum of our business, we are raising our full year 2021 EPS guidance and believe AbbVie is very well positioned for the long term.” ” Second-Quarter Results. Worldwide net revenues were $13.959 billion, an increase of 33.9
today announced new data from its expansive neuroscience portfolio will be presented at the 2021 American Academy of Neurology (AAN) Annual Meeting, to be held virtually from April 17-22. . Key AbbVie abstracts and presentation details for the 2021 AAN Annual Meeting program are outlined below. Abstract Title. Presentation Details.
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.
Approval is based on positive Phase 3 data demonstrating improvements in key disease burden measures and establishing its safety profile Nexviazyme specifically targets the M6P receptor, the key pathway for enzyme replacement therapy, to effectively clear glycogen build-up in muscle cells. Targeted delivery to clear glycogen in muscle cells.
Dylan Verden of KIF1A.ORG summarizes newly published KIF1A-related research and highlights progress in rare disease research and therapeutic development. But the opposite relationship is just as important: knowing which diseases are associated with a symptom means earlier diagnosis, and an earlier search for other potential symptoms.
Dylan Verden of KIF1A.ORG summarizes newly published KIF1A-related research and highlights progress in rare disease research and therapeutic development. Like hereditary spastic paraplegia, CMT is an umbrella disorder, associated with mutations in over 140 genes, but many diagnosed patients do not know their disease-causing mutation.
OCREVUS data show its consistent benefit on slowing disease progression in relapsing multiple sclerosis (RMS) and primary progressive MS (PPMS). Data for ENSPRYNG in neuromyelitis optica spectrum disorder (NMOSD) reinforce safety and efficacy, including in patients with concomitant autoimmune diseases (CAIDs). Following U.S.
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