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A medical breakthrough could result in the first treatment for rare but serious diseases in which genetic defects disrupt cellular energy production. Researchers have identified a molecule that helps more mitochondria function properly.
THURSDAY, March 7, 2024 -- Some acne treatments may may banish blemishes but carry hidden dangers: A new report reveals high levels of the carcinogen benzene can form in products that contain the zit-fighting ingredient benzoyl peroxide.According.
Food and Drug Administration (FDA) plays a pivotal role in fostering the development of treatments for rare diseases through its Orphan Products Grants Program. Each year, FDA selects a limited number of clinical trials to fund to help sponsors pursue development of medical products for rare diseases and advance their field.
Over the years, Ive had the privilege of working on more than 25 different products, Bock shared. In this model, once a product is produced and delivered to the clinical pharmacy, the clinical team takes over the trial with little to no interaction with the manufacturing team.
Inhaled combination products (ICP) have emerged as a significant advancement in the treatment of respiratory diseases such as asthma, chronic obstructive pulmonary disease (COPD), and other pulmonary conditions. However, these products present unique challenges from a Chemistry, Manufacturing, and Controls (CMC) standpoint.
Researchers have now developed a molecular treatment that significantly enhances the efficiency and durability of perovskite solar cells. Their breakthrough will potentially accelerate the large-scale production of this clean energy.
Executives at the biotech say they’re trying to get ahead of the payer and production challenges that will face their gene editing treatment exa-cel, which is now under FDA review.
A gene encoding a protein linked to tau production -- tripartite motif protein 11 (TRIM11) -- was found to suppress deterioration in small animal models of neurodegenerative diseases similar to Alzheimer's disease (AD), while improving cognitive and motor abilities, according to new research.
This exclusive interview with Dr Sharon Benzeno, Chief Commercial Officer, Immune Medicine at Adaptive Biotechnologies, unveils some ground-breaking research on T- cell therapy for cancer , which has seen the first TCR-based therapeutic candidate progress to clinical development, offering promising advancements in innovative cancer treatments.
The Dark Side of Innovation: How Companies Use Drug Patents to Block Competitors As we celebrate the breakthroughs in medical research and the development of life-saving treatments, it's essential to acknowledge the complex landscape of pharmaceutical innovation. One of the most effective tools in their arsenal is the use of drug patents.
There are over 7,000 rare diseases affecting more than 30 million people in the United States, and despite the FDA's approval of over 600 treatments for rare diseases since signing the Orphan Drug Act into law in 1983, most rare diseases still do not have a treatment.
Most of these conditions are genetic in origin and the majority have no effective treatment. That includes countless rare peripheral diseases, including many for which there are currently no treatments. Boosting autophagy won’t be the only road to new treatments for rare genetic diseases but it is a vital one to take.
High-Throughput Screening: Modern Technology Meets Natural Products Advanced technologies now allow researchers to rapidly test thousands of natural compounds against specific disease targets. The development of Taxol involved multiple patents, including those for the isolation method, synthetic production, and various formulations.
This study investigates the effects and mechanisms of combining Polyphyllin VII (PPVII) with Docetaxel (DTX) in the treatment of prostate cancer (Pca). This study reveals the synergistic effects of combining PPVII with DTX and provides a reference for overcoming DTX resistance in clinical Pca treatment.
The Expanding Role of mRNA in Cancer Therapy One of the most exciting applications of mRNA therapeutics lies in cancer treatment, where leveraging the immune system to target tumors offers a novel approach. These findings underscore the potential of mRNA to redefine cancer treatment by offering precise, patient-specific therapies.
The FDA’s decision to turn back the drugmaker’s new insulin product narrows Novo’s lead in the U.S. over Eli Lilly, which has a similar diabetes treatment in late-stage testing.
Patients with inflammatory bowel disease (IBD) report using cannabis to manage their symptoms, despite little data to support the use of cannabis or cannabis products to treat the disease. Taken together these data suggest that high CBG hemp extracts may offer a novel treatment option for patients. Cannabis sativa L.
We previously reported that short-term treatment with SLO at relatively high concentrations (10-1000 ng/mL) diminished acetylcholine-induced, endothelial-dependent relaxation in a concentration-dependent manner. In this study, treatment of rat aorta with endothelium with SLO (0.1-10 In vivo SLO treatment (46.8
Nuclear power is considered one of the ways to reduce dependence on fossil fuels, but how to deal with nuclear waste products is a concern. Radioactive waste products can be turned into more stable elements, but this process is not yet viable at scale.
Abstract The natural products plinabulin, docetaxel, and vinblastine are microtubule targeting agents (MTAs). They have been used alone or in combination in cancer treatment. However, the exact nature of their effects on microtubule (MT) polymerization dynamics is poorly understood.
Molecular-level biochemical assays like transcriptomics, genomics and proteomics have emerged as valuable tools for identifying potential targets in cancer treatment through deep cyclic inhibition (DCI). How does the DCI mechanism compare to the design of other drugs for cancer treatment?
2] The label also has a warning about liver toxicity and the need to monitor liver blood tests at baseline and then monthly for at least six months after initiating treatment with fitusiran or after a dose increase of fitusiran. [2] Fitusiran 1711.0g/mol, 1] It is an antithrombin-directed small interfering ribonucleic acid. [1] 26 March 2025.
8 With the widespread use of induced pluripotent stem cells (iPSCs), organoids can now be generated from the skin or blood cells of both healthy volunteers and disease patients, opening the door to studying various diseases and creating individually customised potential treatments. Kidney repair is one area of urgent need. Nat Rev Cancer.
A combination of network pharmacology and invitro validation was used to reveal the potential targets and molecular mechanisms of resveratrol for CRS treatment. ABSTRACT Resveratrol (RES) is a polyphenolic antioxidant derived from different plant products, which has anti-inflammatory and antioxidative stress effect.
The 8 -THC treatment (30 mg/kg) reduced paw swelling and qualitative signs of arthritis. Significance Statement Despite increasing use of cannabis products, the potential effects of minor cannabinoids are largely unknown. These data support the development of novel cannabinoid treatments for inflammatory arthritis.
A Key contributing factor to this significant growth prediction is the accelerating legalization of cannabis products and the growing acceptance of its use in the medical field and consumer goods.
Susceptibility or risk biomarkers can detect the likelihood of a patient developing a disease or medical condition, which is crucial for treatments that are most effective before the onset of symptoms. A biomarker is a measurable indicator of a biological process, disease state, or response to a treatment.
While most biologically active macrocycles are peptides or natural product-derived , ring-closing metathesis (RCM) reactions have contributed to greater accessibility of unnatural macrocycles in medicinal chemistry. Glecaprevir, or ABT-493, was approved in combination with pibrentasvir (Mavyret) for the treatment of Hepatitis C (HCV) in 2017.
Here we explore the evolution and impact of market exclusivity policies in the EU and US, highlighting their role in fostering innovation and accessibility in rare disease treatment. This was a turning point, igniting a wave of research and development into treatments for rare diseases that had previously been neglected.
Despite their exciting potential, the smooth operation of cell therapy development trials requires extraordinary orchestration, perfectly aligning the product and patient journeys. Delivering this cellular drug product into the patient’s body, followed by intensive medical oversight.
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Casgevy, the commercial product formerly known as exa-cel, is administered by taking stem cells out of a patient’s bone marrow and editing a gene in the cells in a laboratory, with the modified cells then infused back into the patient after conditioning treatment to prepare the bone marrow. In June 2023, the U.S.
A recent analysis of all relevant published studies reveals a lack of evidence to determine the dosing, safety, and efficacy of medical marijuana or cannabis-containing products for managing symptoms experienced by children with cancer. Although treatments for childhood […]
Despite significant progress, there are more than 6,500 rare diseases without a single approved treatment. There are so many patients suffering from diseases that have no treatments. We are committed to supporting reimbursement and exploring ways to increase patient access to treatments. “We
Written by Susannah Sadler and Stacy Grieve Launching a treatment for inflammatory diseases can be a make-or-break endeavor. A robust evidence base and a compelling value story are crucial to success, but current challenges and unexpected bumps in the road will require creative solutions.
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