Latest news with #HLH
Business Wire
16-05-2025
- Health
- Business Wire
Neurogene Announces Evidence-Based Monitoring and Treatment Intended to Reverse Rare Hyperinflammatory Syndrome Associated with High-Dose AAV
NEW YORK--(BUSINESS WIRE)--Neurogene Inc. (Nasdaq: NGNE), a clinical-stage company founded to bring life-changing genetic medicines to patients and families affected by rare neurological diseases, today announced an oral presentation at the American Society of Gene and Cell Therapy (ASGCT) Annual Meeting describing a monitoring and treatment algorithm intended to reverse the rare, severe hyperinflammatory syndrome hemophagocytic lymphohistiocytosis (HLH) that has been associated with systemic exposure to high doses of adeno-associated virus (AAV) gene therapy (>1E14 vg/kg). The 1E15 vg dose level Neurogene is moving forward in its Phase 1/2 trial of NGN-401 gene therapy for Rett syndrome translates into the E13 vg/kg range, and the Company is not aware of any case of HLH ever being reported at this dose level in AAV gene therapy. 'We appreciate the opportunity that ASGCT is providing for us to share findings related to HLH and the evidence-based recommendations for how to monitor, detect and treat this rare, hyperinflammatory syndrome in the context of AAV gene therapy,' said Rachel McMinn, Ph.D., Founder and Chief Executive Officer of Neurogene. 'As we connect with the gene therapy community to provide this information, our hope is that it spurs adoption of the early monitoring and treatment algorithm and increases propensity for sharing trial findings from which others can learn.' Early monitoring and prompt treatment have proven effective in reversing the course of HLH in the setting of higher-dose AAV gene therapy (>1E14 vg/kg) 1,2. Neurogene incorporated the following monitoring and treatment protocol into its Phase 1/2 clinical trial of NGN-401 for Rett syndrome despite the use of a lower dose in the E13 vg/kg range: Implemented daily monitoring for the following in the first week post-dosing: elevated ferritin levels, fever, and falling blood counts (cytopenia), also referred to as the three Fs 3; over 90% of patients with HLH presented with these three initial signs seen in the HLH-2004 study 4 on which current HLH treatment guidelines are based Included HLH treatment algorithm in the trial, which includes high-dose corticosteroids as first-line treatment, and anakinra, the IL-1 receptor agonist, as second-line treatment 1 Galletta et al. J Clin Pharm Ther. 2022;47(9):1478-1481. 2 Byrne et al. Mol Ther. 2022;30(12):3503-3504. 3 Cox et al. Lancet Rheumatol. 2024;6:e51–62. 4 Bergsten et al. Blood. 2017;30(25)2728-2738. About Neurogene The mission of Neurogene is to treat devastating neurological diseases to improve the lives of patients and families impacted by these rare diseases. Neurogene is developing novel approaches and treatments to address the limitations of conventional gene therapy in central nervous system disorders. This includes selecting a delivery approach to maximize distribution to target tissues and designing products to maximize potency and purity for an optimized efficacy and safety profile. The Company's novel and proprietary EXACT™ transgene regulation platform technology allows for the delivery of therapeutic levels while limiting transgene toxicity associated with conventional gene therapy. Neurogene has constructed a state-of-the-art gene therapy manufacturing facility in Houston, Texas. CGMP production of NGN-401 was conducted in this facility and will support pivotal clinical development activities. For more information, visit Cautionary Note Regarding Forward-Looking Statements Statements in this press release which are not historical in nature are intended to be, and hereby are identified as, forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995. These statements may discuss goals, intentions and expectations as to future plans, trends, events, results of operations or financial condition, or otherwise, based on current expectations and beliefs of the management of Neurogene, as well as assumptions made by, and information currently available to, management of Neurogene, including, but not limited to, statements regarding: the safety, tolerability and efficacy of NGN-401; the effectiveness of the monitoring and treatment protocol for HLH in Neurogene's Phase 1/2 clinical trial of NGN-401; and the ability to reverse cases of AAV-related HLH when following this protocol. Forward-looking statements generally include statements that are predictive in nature and depend upon or refer to future events or conditions, and include words such as 'may,' 'will,' 'should,' 'would,' 'expect,' 'anticipate,' 'plan,' 'likely,' 'believe,' 'estimate,' 'project,' 'intend,' 'on track,' and other similar expressions or the negative or plural of these words, or other similar expressions that are predictions or indicate future events or prospects, although not all forward-looking statements contain these words. Forward-looking statements are based on current beliefs and assumptions that are subject to risks, uncertainties and assumptions that are difficult to predict with regard to timing, extent, likelihood, and degree of occurrence, which could cause actual results to differ materially from anticipated results and many of which are outside of Neurogene's control. Such risks, uncertainties and assumptions include, among other things, the risks and uncertainties identified under the heading "Risk Factors" included in Neurogene's Annual Report on Form 10-K for the year ended December 31, 2024, filed with the Securities and Exchange Commission (SEC) on March 24, 2025, Neurogene's Quarterly Report on Form 10-Q for the quarter ended March 31, 2025, filed with the SEC on May 9, 2025, and other filings that the Company has made and may make with the SEC in the future. Nothing in this communication should be regarded as a representation by any person that the forward-looking statements set forth herein will be achieved or that the contemplated results of any such forward-looking statements will be achieved. Forward-looking statements in this communication speak only as of the day they are made and are qualified in their entirety by reference to the cautionary statements herein. Except as required by applicable law, Neurogene undertakes no obligation to revise or update any forward-looking statement, or to make any other forward-looking statements, whether as a result of new information, future events or otherwise.
Medical News Today
12-05-2025
- Health
- Medical News Today
NK cell leukemia (ANKL): Symptoms, causes, and treatment
Symptoms Causes and risk factors Diagnosis Summary ANKL is a rare and aggressive type of blood cancer affecting a type of white blood cell called natural killer (NK) cells. In ANKL, the NK cells are abnormal and multiply out of control, crowding out other healthy blood cells. This leads to serious and life threatening complications. Aggressive natural killer (ANK) cells belong to a group of white blood cells called 'lymphocytes' which play a vital role in the body's immune system. NK cells in particular target and destroy disease-causing pathogens, and detect and control the early signs of cancer. In ANKL, the NK cells are unable to perform their usual functions. The accumulation of cancerous or 'neoplastic' NK cells in the body leads to serious and life threatening can present differently from person to person. However, the most common signs and symptoms include: fever night sweats unintentional weight loss swelling of the lymph nodes People with ANKL commonly present with a rare and life threatening inflammatory condition called 'fulminant hemophagocytic lymphohistiocytosis (HLH). This condition occurs as a result of immune system overactivation, and can lead to multiple organ failure. Common HLH triggers include: infection, with Epstein-Barr virus (EBV), which is the most common cause acute illness cancer The two most common clinical features of HLH are fever and enlargement of the spleen (splenomegaly). The latter may cause symptoms such as: abdominal discomfort pain in the upper left abdomen referred pain in the upper left shoulder abdominal bloating feeling full after eating very little (early satiety) weight loss Due to the rarity of NKL, scientists are still working to determine its causes and risk factors. While anyone can develop ANKL, the average age at disease onset is 40 years, and the disease is more common among people of Asian ethnicity. EBV infection is a risk factor for ANKL. As the Centers for Disease Control and Prevention (CDC) explains, EBV is among the most common human viruses in the world, and most people develop EBV at some point in their lifetime. Children rarely experience symptoms, while teenagers and adults may experience symptoms for several weeks or months. Once a person has had EBV, the virus becomes inactive or 'latent' in their body. However, the virus can express viral proteins that disrupt cell functioning, suppress cell death, and trigger uncontrolled cell growth. This, in turn, can increase the risk of certain cancers. Cases of ANKL that are associated with EBV infection are referred to as 'EBV-positive'. ANKL can also develop in people who have never had EBV. Doctors refer to this type of ANKL as 'EBV-negative'. Unlike EBV-positive ANKL, EBV-negative ANKL occurs most often in older adults and arises equally among people of Asian and non-Asian ethnicity. However, EBV-positive and EBV-negative ANKL appear equally aggressive in terms of their clinical onset and disease course. Doctors may have difficulty accurately diagnosing ANKL due to the following: The disease is rare and has a rapid onset. The disease has no specific pathologic features and shares symptoms with many other conditions. The disease has no standard immunophenotypic features, meaning that the diseased cells do not show any features specific to ANKL, such as specific antigens or markers on their surface. ANKL can be particularly difficult to diagnose in its early stages due to the small number of cancerous NK cells in the bone marrow. According to some studies, this number may be as low as 5% or even less. As the disease progresses, the number of cancerous NK cells in the bone marrow rapidly increases. As such, the reviewers recommend that doctors conduct multiple blood or bone marrow specimens at various time points to check for the disease. Once doctors have retrieved a blood or bone marrow sample, they will send it to a lab for analysis using flow cytometry. This analysis helps determine the types of cells, their characteristics, and the presence of tumor markers. » Learn more:What tests can diagnose leukemia? Treatment for ANKL typically involves chemotherapy, radiation therapy, or a combination of the two. Large-scale clinical studies are necessary to determine the optimal treatment approach. A person with ANKL can consider talking with their doctor about the possibility of joining clinical trials. Combined radiation therapy and chemotherapy regimens incorporating the chemotherapy drug 'L-asparaginase' appear most effective for treating ANKL. An example of such a regimen is the 'SMILE' regimen, which consists of the following drugs: dexamethasone methotrexate ifosfamide L-asparaginase etoposide » Learn more about leukemia treatment. The outlook for ANKL is typically very poor , even among individuals on the more successful treatment regimens, such as SMILE. In a small, older study from 2016, only 50% of 13 individuals on the SMILE regimen experienced a partial or complete response to treatment, with only 27.8% experiencing a complete response to treatment. Eight individuals then received a follow-up blood and bone marrow transplant called 'hematopoietic stem cell transplantation (HSCT)', which involves implanting stem cells to promote healthy blood cell production. Six individuals remained alive following the procedure. A 2017 study of 21 people with ANKL also demonstrated improved survival rates among those who received HSCT following their initial treatment. Of the 17 individuals who received an L-asparaginase-containing regimen, 14 experienced a complete treatment response prior to HSCT. At 25-month follow-up, the 2-year progression-free survival (PFS) was 20%, and the overall survival (OS) was 24%. The term 'PFS' refers to the length of time a person lives with a disease without it worsening, while the term 'OS' refers to the average length of time a person lives following their initial diagnosis. Aggressive natural killer cell leukemia (ANKL) is a rare and aggressive type of blood cancer that affects white blood cells called 'natural killer (NK) cells'. In ANKL, the NK cells are abnormal and multiply out of control, crowding out other healthy blood cells in the blood and bone marrow. This leads to serious and life threatening complications. The Epstein-Barr virus (EBV) is a risk factor for ANKL. Cases of NKL related to EBV are referred to as 'EBV-positive', while those unrelated to EBV are referred to as 'EBV-negative'. Due to the rarity and aggressiveness of ANKL, doctors have yet to establish a standard treatment approach. However, treatments incorporating the chemotherapy drug L-asparaginase appear to be the most effective. The current outlook for ANKL is poor. Research is ongoing to help determine more effective treatments. Leukemia Blood / Hematology Cancer / Oncology Medical News Today has strict sourcing guidelines and relies on peer-reviewed studies, academic research institutions, and medical journals and associations. We only use quality, credible sources to ensure content accuracy and integrity. You can learn more about how we ensure our content is accurate and current by reading our editorial policy. About Epstein-Barr virus. (2024). Chapman J, et al. (2023). Splenomegaly. Eissmann, P. (n.d.). Natural killer cells. El Hussein S, et al. (2020). Aggressive NK cell leukemia: Current state of the art. Hamadani M, et al. (2017). Allogeneic hematopoietic cell transplantation for aggressive NK cell leukemia. A center for international blood and marrow transplant research analysis. Hines M, et al. (2018). Diagnosis, treatment, and management of hemophagocytic lymphohistiocytosis in the critical care unit. Critical care of the pediatric immunocompromised hematology/oncology patient. Jung K. S, et al. (2016). L-asparaginase-based regimens followed by allogeneic hematopoietic stem cell transplantation improve outcomes in aggressive natural killer cell leukemia. Shannon-Lowe C, et al. (2017). Epstein-Barr virus-associated lymphomas. Spaner C, et al. (2024). Case report: Aggressive natural killer cell leukemia and refractory hemophagocytic lymphohistiocytosis in an adolescent. Sumbly V, et al. (2022). Aggressive natural killer cell leukemia: A brief overview of its genomic landscape, histological features, and current management. Tse E, et al. (2017). The diagnosis and management of NK/T-cell lymphomas.
Yahoo
25-03-2025
- Lifestyle
- Yahoo
Home Living Handbook, Formerly a Men's Journal 'Pursuits' Channel, Launches as Independent Destination for Everything Home, on Par with The Spruce, Martha Stewart, Family Handyman
The new home improvement / home maintenance / DIY site led by veteran home enthusiast writer/editor, Emily Fazio, aims to provide homeowners & renters with expert-led content inspiring a home suitable for them, designed for them, and adaptable to them NEW YORK, March 25, 2025 /PRNewswire/ -- Today, Home Living Handbook (HLH), a trusted online authority with +6M views, launched as its own independent digital destination at Leveraging insights from home experts spanning construction, real estate, design, maintenance, home efficiency and technology & beyond, the new site will inform and inspire homeowners on how to tackle the fun and not-so-fun projects in, on, around and outside the home. Formerly one of more than twenty dedicated "Pursuits" channels premiered in the fall of 2023, the high-performing HLH has migrated its content from The Arena Group-owned site, establishing its own domain complete with new look, branding, categories and robust subcategories. In an attempt to capture niche readers, tapped Emily Fazio, a 15+ year home improvement and DIY writer, to build HLH from the ground up -- including sourcing a team of consultants, writers, and overseeing all content development. Fazio's following in the home space stems from her experience writing for HGTV, the former and her personal blog, a website she started when she became a homeowner in 2009. Through her work, she has developed authority and gained rapport with national and global home and tool brands, as well as a network of industry experts. Recognizing its readers' appreciation for longform content -- a driving force behind HLH's 300% YoY growth -- the new website will continue to prioritize in-depth pieces while allowing for deeper exploration of micro-niche topics. In addition to HLH's hero categories: lawn care, home maintenance, cleaning, smart home technology, seasonal trends, sustainability, and home energy efficiency, new categories like parents and pet-owners have been built out to appeal to a broader audience. Articles like "Watch For These Homeowner and Homebuyer Trends in the Next Year" and "How to Insulate Your Basement Rim Joists," have amassed a following of 4.3M active HLH users and 600K returning users since the brand's initial debut. The launch of HLH as a standalone site reinforces consumer's departure from relying on brand name, legacy media, and the power digital natives hold to deliver highly-specialized, on-demand content. Expanding upon its focus of guiding readers through the good, bad, and ugly -- (and pretty!)-- aspects of home ownership and everyday living, HLH will continue to deliver expert insights, home trends, product reviews, and DIY inspiration for homeowners, do-ers, renters, and enthusiasts alike. Press Contact:Samantha Perriello392319@ About Home Living Handbook: Since 2023, Home Living Handbook has provided practical, expert-backed advice, resources, and inspiration to help you create, maintain, and enjoy your home—inside and out. Whether deciding between a DIY project or hiring a contractor, looking for real estate insights, or seeking industry expert home maintenance tips, Home Living Handbook's trusted, actionable advice has helped millions of readers make informed decisions every step of the way. With new content posted daily, Home Living Handbook helps homeowners, renters, and DIY enthusiasts find the guidance they need to renovate their spaces, tackle home repairs, enhance their gardens, and elevate their interiors. Our extensive library of articles include in-depth industry expert interviews, step-by-step guides, and practical solutions to make every home project a success, as we believe your home should be a reflection of you. View original content to download multimedia: SOURCE Home Living Handbook Sign in to access your portfolio
BBC News
24-03-2025
- Entertainment
- BBC News
Inverness Castle ticket prices announced
Entry fees for Inverness Castle have been former sheriff court and prison is being redeveloped as a visitor attraction at a cost of about £30m, and is due to open later this Life Highland (HLH), which will operate the site, has released the ticket prices which range from the cheapest - £14 for a child - to up to £65 for a family of two adults and three said deals would be available to Highland residents. The redevelopment has received funding from the £315m Inverness and Highland City Region the Inverness Castle Experience, it will have an interactive "journey" taking visitors through the history of the Highlands. How much will tickets cost? HLH said tickets would be cheaper if booked online in costs include:Adult - £20 (online)/£22Seniors - £18/£20Child - £14/£16Family (two adults and two children) - £46/£54Family (two adults and three children) £55/£65HLH said Highland residents who pay for its High Life Highland card would have access to more booking options, and 10% discount on food and drinks after purchasing full-price tickets. Ian Brown, a councillor and co-chairman of Inverness Castle Delivery Group, has welcomed ticket deals for Highland added: "The Inverness Castle Experience is set to become a key landmark in the region and ensuring that our Highland communities can enjoy it throughout the year is a great move. "This initiative encourages local engagement and fosters pride in our heritage and Highland stories."The Scottish Courts and Tribunal Service moved out of the castle in 2020, and Highland Council has been leading the month, public agency Highlands and Islands Enterprise (HIE) put an extra £863,000 towards the project. What is the castle's history? The sandstone Inverness Castle was built in 1836 to plans drawn by architect William was constructed on a mound overlooking the city and the River Ness. In 1848, a building known as the North Block was added and served as a a castle had occupied the site from possibly as far back as the 11th the centuries, the fortification fell under the control of the forces of Edward I, Robert the Bruce and James I, II and 1562, it was attacked and damaged by soldiers loyal to Mary, Queen of Scots, before it was almost destroyed by Royalist troops in the Prince Charlie's forces blew the castle up in 1746 to prevent it from falling into the hands of government was said that a French sergeant, who had brought his poodle with him to Scotland, set off the soldier was caught in the blast, and his body was blown across to the opposite side of the River Ness. His dog survived.
BBC News
19-03-2025
- Health
- BBC News
Newport: Baby with rare life-threating disorder finds donor
A four-month-old baby diagnosed with a rare blood disorder has found a donor and had a stem cell transplant that could save her Edwards-Raymond, from Newport, has hemophagocytic lymphohistiocystosis (HLH) which can be life-threatening if left untreated or not diagnosed aunt, Stephanie Curtis, said the transplant had given her niece a "second chance of life".The stem cells were not an exact match but the family were "hoping and praying" that that her body reacts well and were now waiting for the results. HLH is an immune deficiency disorder which happens when certain types of white blood cells, known as histiocytes and lymphocytes, become overactive and attack the were 1,674 people with HLH diagnosed in England between 2003 and 2018, according to a study published in 2022. Following her diagnosis, Dolcie-Mae was taken to the Royal Victoria Infirmary in Newcastle upon Tyne, where she was receiving specialist family had appealed for people between the ages of 16 and 30 to join the Anthony Nolan stem cell register to find a Curtis said the donor was found "a couple of weeks ago" which led to Dolcie-Mae's cells being replaced."The lab had to get all the cells ready, take the bad ones out and give the good one's to Dolcie-Mae. I believe it was between five and seven days of chemotherapy to get rid of her cells," said Ms and her mother Courtney-Jade remain at the hospital while the rest of the family are in their hometown of Newport."On the seventh or eighth day she had the transplant with the donors cells. I believe they were from Germany." Ms Curtis said it would be "100 days from the transplant date" as the hospital continued to do regular tests to measure the number of cells in her blood."If her levels stay up, which hopefully they do, they think that the transplant will be successful."Ms Curtis said the family were "keeping everything crossed" that it would be a successful match."We can only hope really that this has worked for Dolcie-Mae." Ms Curtis said that as the donor was not a complete match it "won't be the best chance, but she will have a chance"."We're all really happy now for Courtney and Dolcie-Mae. She's got all the support she can have with her friends and family."Ms Curtis also heaped praised on the doctors and nurses who have cared for Dolcie-Mae since she was taken to the Royal Victoria added: "We appreciate every single thing they do." The whole community has pulled together to support Dolcie-Mae, according to Ms Curtis."Until you go through something like this, you don't realise how close-knit the community is," she family were planning a fundraiser at the weekend to raise awareness about stem cell a social media post the charity said that the mismatched stem cell transplant would "be her best treatment option" as there were no matches worldwide on the current register.
