A uniQure gene therapy for Huntington’s disease has interim clinical data showing an 80% slowing of disease progression. An accelerated approval pathway is one of the topics the company wants to discuss with the FDA. By Frank Vinluan on July 09, 2024A uniQure gene therapy for Huntington’s disease has interim data showing the one-time treatment slows progression of this rare neurodegenerative disorder that has no FDA-approved therapies. Based on these encouraging data, the company said Tuesday it now plans to discuss with regulators the potential for a faster clinical and regulatory path forward. Huntington’s stems from a genetic mutation that leads to abnormal versions of the huntingin protein. The disease leads to motor dysfunction, behavioral changes, and cognitive decline. The uniQure gene therapy, code-named AMT-130, uses an engineered virus to deliver microRNA that silences the huntingin gene and the toxic protein fragment that drives the disease. Amsterdam-based UniQure is evaluating ...
Organizer: IPHEB Company, Russia Date: April 8-10, 2025 Venue: Peterburgskoye sh. 64/1, St. Petersburg, Russia Exhibition hall:ExpoForum Convention and Exhibition Centre Product range: Pharmaceutical raw materials, intermediates, and formulations; antibiotic, vitamin, and amino acid-based raw materials for pharmaceuticals and veterinary drugs; Food ingredients and additives, feed additives; Pharmaceutical machinery, laboratory equipment, packaging equipment, materials, and new technologies; Contract manufacturing services, biomedical products Exhibition Introduction: The International Pharmaceutical and Healthcare Exhibition (IPhEB Russia), held in St. Petersburg, Russia, is the most influential exhibition for pharmaceutical raw materials in the country. It presents a unique opportunity for businesses in the pharmaceutical industry of Russia and the Commonwealth of Independent States (CIS) to conduct business and forge partnerships, making it the leading pharmaceutical event in the region.
Progressive supranuclear palsy (PSP) is a severe neurological disorder classified under frontotemporal dementia (FTD) that impairs cognition, movement, and behavior. Thought to be caused by an accumulation of tau proteins that damage and kill cells, PSP presents symptoms such as poor balance with frequent backward falls and difficulties moving the eyes vertically. Often misdiagnosed as Parkinson’s disease, PSP progresses more rapidly and does not respond to Parkinson’s treatments. Most individuals with PSP pass away approximately seven years after symptoms begin, and frequently, the disease is only identified posthumously during an autopsy. Unlike Alzheimer’s, there are no tau scans, blood tests, or MRIs that can conclusively diagnose PSP, a fact that has hampered the development of treatments. Now, the discovery of a unique pattern of proteins in the spinal fluid of patients could lead to earlier diagnosis and the development of new treatments for PSP. In a study published in Neurology ...
Whether it’s more sleep or more exercise, simple lifestyle changes may reduce the risk of dementia. Now, a new program from the University of South Australia in collaboration with research partners at Onkaparinga Council and ACH group is helping older Australians make healthier choices to reduce their risk of dementia. Running over 12 weeks, UniSA’s ‘Small Steps’ program will support people aged 65 years and over to make small, incremental changes to their lifestyle choices to improve sleep, reduce sitting time and increase physical activity. UniSA researcher Dr Catherine Yandell says the program hopes to provide participants with genuine insights into their sleep and movement as it relates to dementia risk. “Maintaining an active and healthy lifestyle is essential to reduce the risk of dementia later in life, yet few older adults meet current activity guidelines, and even fewer continue them in the long term,” Dr Yandell says. “Part of ...
By Mike Hollan The platform is similar to other recent digital platforms in that it is designed to reduce drug costs. One of the main issues impacting the pharmaceutical industry is the cost of medications. Recently, several digital platforms have launched with the mission of providing more competition by providing purchasers with more options for buying drugs. While the most prominent of these platforms have been based on a direct-to-consumer model, there are other platforms that are designed for pharmacies to use as well. PrimeRX announced the launch of a new platform aimed at pharmacies named PrimeRX MARKET.1 This platform will provide users with access to over 40 different drug suppliers, along with the ability to place all of their orders through one platform, and provide research on drug prices. In a press release, PrimeRx CEO Ketan Mehta said, “We are thrilled to introduce PrimeRx MARKET to our solutions portfolio. ...
By Don Tracy, Associate Editor Funding from the Biomedical Advanced Research and Development Authority (BARDA) is expected to support the late-stage development and licensure of a pre-pandemic vaccine targeting the H5 influenza virus. The Biomedical Advanced Research and Development Authority (BARDA) has granted Moderna a $176 million project award under the Rapid Response Partnership Vehicle (RRPV) Consortium, with the goal of expediting the development of mRNA-based pandemic influenza vaccines. Reportedly, the award is expected to support the late-stage development and licensure of a pre-pandemic vaccine targeting the H5 influenza virus, a subtype known for causing severe avian influenza and posing a potential risk to humans.1 “mRNA vaccine technology offers advantages in efficacy, speed of development, and production scalability and reliability in addressing infectious disease outbreaks, as demonstrated during the COVID-19 pandemic,” said Stéphane Bancel, CEO, Moderna, in a press release. “We are pleased to continue our collaboration with BARDA to ...
Only 10% of individuals diagnosed with pancreatic cancer will live beyond two years post-diagnosis. The ability to predict the timing and location of metastases could dramatically transform the treatment approach for pancreatic cancer, especially for those at high risk of metastasis. Now, researchers have employed liver biopsies—a small tissue sample taken for laboratory analysis—to identify cellular and molecular markers that might predict if and when pancreatic cancer will metastasize to the liver or other areas like the lungs. A study conducted by Weill Cornell Medicine (New York, NY, USA) and an international research group suggests that data from liver biopsies obtained at the time of pancreatic cancer diagnosis could inform personalized treatment plans, such as liver-targeted immunotherapies, to preempt the spread of cancer cells. The research team discovered in 2015 that pancreatic cancer cells emit substances that primarily target the liver to create a pre-metastatic niche that promotes the growth ...
Organiser:Informa Markets Time: September 8-10, 2024 Address:El-Nasr Rd, Al Estad, Nasr City, Cairo Governorate Exhibition hall:Cairo International Convention Centre Product range: Vitamins, Hormones, Antipyretic Analgesics, Digestive System Medications, Anti-infective Drugs, Antiparasitic Drugs, Cardiovascular System Medications, Intermediates Pharmaceutical Packaging Materials: PVC/PVDC Laminates, PVC Rigid Sheets, PS Sheets, Anti-Counterfeiting Technology for Packaging, Label Sleeve Technology, Labeling Machines, Packaging Containers, Form-Fill-Seal Machines, Wrapping Machines, Paper Film Packaging Machinery, Bag Packaging Machines, etc. Infusion Equipment Production Lines, Dryers, Water Treatment Equipment, Refrigeration Compressors, Distillation Equipment, Sterilization Equipment, Tablet Presses, Coating Machines About Pharmaconex: Pharmaceutical Ingredients & Technology Exhibition in Cairo, Egypt, is the most renowned exhibition for pharmaceutical ingredients and technology in Egypt. Hosted by the renowned British exhibition company Informa Markets, with extensive experience and strong capabilities in organizing exhibitions, Pharmaconex plays a significant role in exploring the markets of Egypt and Africa.
Early detection of cancer significantly increases the likelihood of successful treatment across various cancer types. In addition to personalized evaluation of therapy benefits and risks, continuous monitoring of treatment success is vital. Traditionally, oncologists rely on imaging technology and invasive procedures such as tissue biopsies, punctures, and endoscopic measures to monitor patients. Now, researchers have further developed an advanced method, a type of liquid biopsy that utilizes blood samples rather than direct tissue sampling for detecting various types of cancer. The technique, developed by researchers at the University of Zurich (UZH, Zurich, Switzerland) and the University Hospital Zurich (USZ, Zurich, Switzerland), involves sequencing and analyzing DNA fragments that circulate in the blood of patients to identify changes specific to particular cancers. The research team examined alterations in the number and length distribution of the DNA fragments, enabling them to distinguish between biologically less and more aggressive metastatic cancers even earlier ...
By Don Tracy, Associate Editor Kisnula is the first amyloid plaque-targeting therapy that allows for stopping treatment upon plaque removal, company says. The FDA has approved Eli Lilly’s Kisunla (donanemab-azbt, 350 mg/20 mL once-monthly injection for IV infusion) to treat adults with early symptomatic Alzheimer’s disease, including mild cognitive impairment and mild dementia with confirmed amyloid pathology. According to the company, Kisunla the first amyloid plaque-targeting therapy that allows for stopping treatment upon plaque removal, potentially reducing costs and infusion frequency. Approval was based on results from the Phase III TRAILBLAZER-ALZ 2 study.1 “Kisunla demonstrated very meaningful results for people with early symptomatic Alzheimer’s disease, who urgently need effective treatment options. We know these medicines have the greatest potential benefit when people are treated earlier in their disease, and we are working hard in partnership with others to improve detection and diagnosis,” said Anne White, EVP, president, Lilly Neuroscience, Eli ...
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