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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: Juliane Mills, Senior Director, Therapeutic Strategy Lead, Rare Disease The rise of patient-led clinical research, particularly in rare disease, represents a significant shift in the clinical trial landscape. Why Is There an Increase in Patient-Led Rare Disease Research?
Imagine being able to create an in vitro replica of a diseased organ to study the molecular mechanism underlying the illness. Now take a step further: envision testing drugs in these organoids to identify the ones that can treat disease safely and effectively without needing to run expensive clinical trials first.
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.
Neuropsychiatric treatment is on the verge of a major transformation. Historically, treatment options have been limited, with patients relying on daily medications that have minimal efficacy and troublesome side effects. “Zelquistinel is a positive modulator of NMDA receptors,” Donello explains.
Sanofi Highlights Robust Pipeline of Innovations in Rare Blood Disorders at ISTH 2025 Sanofi is poised to take center stage at the 33rd Congress of the International Society on Thrombosis and Haemostasis (ISTH), set to be held in Washington, D.C., from June 21 to 25, 2025. In the U.S.,
This unique approach prevents attacks from occurring before they start, offering patients a new opportunity to manage their condition while reducing the immense disease burden. ANDEMBRY, a novel once-monthly subcutaneous treatment that inhibits factor XIIa, is a welcome addition to the HAE treatment landscape,” said Anthony J.
As our understanding of the underlying biology of disease grows more sophisticated, emerging therapies operate on increasingly complex biopathological systems and mechanisms. Monitoring biomarkers can help assess changes in a disease, its level of expression, or the extent of its progression.
While RA therapies targeted to specific inflammatory pathways have emerged, only some patients’ symptoms improve with treatment, emphasizing the need for multiple treatment approaches tailored to different disease subtypes.
mg/mL solution for injection) across the European Union (EU), allowing for extended treatment intervals of up to six months for individuals diagnosed with neovascular (wet) age-related macular degeneration (nAMD) and visual impairment due to diabetic macular edema (DME).
1] [2] Adverse effects The US Food and Drug Administration prescription label for fitusiran contains a boxed warning for thrombotic events (blood clotting) and gallbladder disease (with some recipients requiring gallbladder removal). [2] 2] [3] Names Fitusiran is the international nonproprietary name. [4] Fitusiran 1711.0g/mol,
A New Treatment Option for a Debilitating Joint Tumor TGCT is a rare, debilitating condition that primarily affects young and middle-aged adults, often striking individuals in their prime working years. Pimicotinib: A Promising CSF-1R Inhibitor Pimicotinib is an investigational small molecule developed by Abbisko Therapeutics Co.,
However, researchers lack efficient ways to explore how these genetic variants are molecularly connected to cardiovascular disease, limiting efforts to develop therapeutics. The team found that a key biological mechanism involved in a rare vascular disease may influence CAD risk.
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. ADUHELM is indicated for the treatment of Alzheimer’s disease.
The resulting mixture was stirred at an internal temperature of about 65° C. was further added thereto, and then the resulting mixture was stirred at an internal temperature of 79° C. was added dropwise at an internal temperature of 22° C. The resulting mixture was stirred at an internal temperature of 19° C. for 4 hours.
Abstract This study was designed to explore the protective effect and mechanism of naringin (NG) on radiation-induced heart disease (RIHD) in rats. The results showed that NG treatment significantly attenuated the 20 Gy XR-induced decline of LVEF and LVFS and the elevation of LVIDs.
Drug discovery is a complex and vital field that continually seeks to identify new therapeutic targets and develop effective treatments. Dewpoint’s breakthrough was the realisation that certain condensates are associated with human diseases. Klein notes the importance of Dewpoint’s disease-agnostic approach.
Written By: Derek Ansel, MS, CCRA, Executive Director, Therapeutic Strategy Lead, Rare Disease Given that 80% of rare diseases have a genetic etiology, genetic implications should be addressed at the onset of a clinical program to support trial enrollment.
High-Throughput Screening: Modern Technology Meets Natural Products Advanced technologies now allow researchers to rapidly test thousands of natural compounds against specific disease targets. Artemisinin: Nobel Prize-Winning Malaria Treatment Artemisinin, derived from the sweet wormwood plant, has become a crucial treatment for malaria.
The results, recently published in the International Journal of Molecular Sciences , come from a collaborative effort between researchers at Erlangen University Hospital and Berlin-based biotech firm AudioCure. Importantly, the treatment also appeared to reverse the underlying physiological damage believed to contribute to tinnitus.
This leadership transition arrives at a pivotal moment in Alnylam’s evolution, as the company builds upon the recent commercial success of AMVUTTRA® (vutrisiran) —a landmark treatment for transthyretin amyloidosis with cardiomyopathy (ATTR-CM). He holds a B.A.
Biogen Unveils New Data Underscoring the Long-Term Benefits and Advancements of Nusinersen in Spinal Muscular Atrophy Treatment Biogen , a global leader in neuroscience, has announced compelling new data that reinforces the clinical value and therapeutic potential of nusinersen, a pioneering treatment for spinal muscular atrophy (SMA).
functions to reset the immune system and potentially provide remission for allergic diseases? In allergic diseases, APCs identify and present unknown antigens to the immune system. This can lead to inadequate long-term effectiveness with low rates of disease remission. They also require frequent administration.
It will also cover recent advancements in broadening their application, including innovative approaches to ensure their safety and efficacy, the use of targeted delivery to reach disease-relevant tissues as well as success stories in bringing therapeutic oligonucleotides to the clinic.
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. flash (powering the free Google App tier) and Claude Sonnet 3.5.
Now an international team led by the Hudson Institute of Medical Research has found a way to determine which species are important and how they interact to create a healthy microbiome. The more diverse species in your gut, the better it is for your health. Credit: Hudson Institute of Medical Research The more diverse species […]
Their approach to kidney disease demonstrates both scientific excellence and a strong commitment to addressing unmet medical needs. Current treatments offer limited efficacy and are often accompanied by severe side effects. NXI Therapeutics brings not only strong science, but a clear strategy for international growth.
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.&
The trial met both its primary and key secondary endpoints, signaling a potential breakthrough in a condition with few, if any, effective treatment options. It is characterized by early-onset, treatment-resistant seizures, beginning as early as six weeks of age, and profound global neurodevelopmental impairment.
18, 2021 /PRNewswire/ — Aruvant Sciences, a private company focused on developing gene therapies for rare diseases, and Lonza announced today their agreement in support of ARU-1801, Aruvant’s one-time investigational gene therapy for sickle cell disease (SCD).
NEW YORK , Jan.
Liso-cel achieved high, lasting response rates in patients with relapsed or refractory marginal zone lymphoma, underscoring its potential to significantly improve patient outcomes in this hard-to-treat disease,” said M. Lia Palomba, M.D. , This data signals a new opportunity for transformation in the standard of care for these patients.”
TEV-56278 is a first-in-class anti-PD1-IL2 ATTENUKINE therapy designed by Teva’s internal team of innovative scientists. This innovative approach stimulates and strengthens their activity against the tumor while reducing the collateral damage — the side effects typically related to IL-2 — frequently seen with less-targeted treatments.
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.
This shift in focus is especially critical in toxicology, where accurate target analysis plays a vital role in identifying toxic effects and ensuring patient safety, particularly as the field transitions from traditional drugs to the promising realm of biotherapeutics, especially for rare diseases.
Researchers at the Allen Institute for Cell Science , a division of the Allen Institute in Seattle, US, developed a unique mathematical framework to uncover internal organisation of cellular structures in human induced pluripotent stem cells (hiPSCs) while taking account of the wide range of variation that naturally occurs in cell shapes.
The study, which is the largest to date that focuses exclusively on this patient population, shows high overall response rates (ORR) alongside deep and durable responses, offering much-needed hope for individuals whose disease typically carries a poor prognosis. Head of Myeloma Unit at Tel-Aviv Sourasky Medical Center in Israel.
FDA Approves Dupixent as the First and Only Targeted Therapy for Adults with Bullous Pemphigoid In a groundbreaking development for patients suffering from a rare and debilitating autoimmune skin disease, the U.S. A hallmark of the disease is its type 2 inflammatory profile , which contributes to its chronicity and relapsing nature.
Challenge to Support an Innovative Solution That Addresses Disconnect in Perception of Psoriatic Disease Severity and Undertreatment Documented in Multinational UPLIFT Survey UPLIFT Data Presented at WPPAC 2021 Report Substantial Disease Burden for People Living With Psoriasis and Psoriatic Arthritis.
With 20-25% of the global population affected by metabolic problems, finding effective treatments is crucial for cardiovascular and metabolic (CVM) conditions. Traditional study designs feature predetermined sample sizes, assessments, and analyses that must remain in place over long treatment durations.
More than just a digital upgrade, the website represents the company’s transformation from a pioneering Japanese biotech startup into a global force in the field of rare and genetic diseases. At JCR, we are committed to advancing science and delivering on the treatments for patients with unmet needs around the world,” said Mr. Ashida.
In the current study undertaken by an international team of researchers, co-led by Dr William Reay, we used the genetics of retinol itself as a way to proxy its effect in thousands of traits, including brain structure, vision, and the immune system through causal modelling.
Advances in research and development and the utilisation of combination treatment strategies are revolutionising the haematology care landscape, but more must be done to address patients’ individual needs. 2 It is this complexity that necessitates powerful, targeted combination therapies.
In cases where control or treatment groups show large variability, this could reflect true biological responses to a treatment, and removing such data could reduce the accuracy of the analysis. It is crucial during this phase to strike a balance between removing erroneous data and preserving meaningful biological variability.
The FDAs expedited programs are intended to facilitate and advance the development and review of new drugs and biologics to address unmet medical needs in the treatment of serious or life-threatening conditions, such as cancer. first or second line setting), considering clinical, scientific, regulatory, and operational factors.
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