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Malaysian Reserve
26-05-2025
- Health
- Malaysian Reserve
Rett Syndrome Market Sees Momentum with Novel Gene and Protein-Targeted Therapies
Rett syndrome market dynamics are expected to undergo significant shifts as more targeted therapies, including gene therapies and pharmacological interventions, enter clinical development and regulatory approval stages. Additionally, increased awareness, improved diagnostic methods, and collaborative efforts within the healthcare community may drive Rett syndrome market expansion and accessibility to treatments for affected individuals. LAS VEGAS, May 26, 2025 /PRNewswire/ — Rett syndrome is a rare genetic neurological condition that typically emerges in infancy and primarily affects girls, though occasional cases have been observed in boys. The disorder causes profound impairments that impact nearly all aspects of a person's life. It is the second leading cause of severe intellectual disability in females. During the stage of developmental regression, many individuals with Rett syndrome also meet the diagnostic criteria for autism spectrum disorder (ASD). The condition mostly affects females because it is linked to mutations on the X chromosome. Females have two X chromosomes, which can provide some genetic compensation, whereas males, who have only one X chromosome, are less likely to survive if the MECP2 gene is mutated. There is no known cure for Rett syndrome, and treatment focuses on symptom management through a comprehensive, multidisciplinary approach. Care often involves coordination among various specialists, including pediatricians, pediatric neurologists, gastroenterologists, speech therapists, psychiatrists, nutritionists, and others, to address the child's complex needs. Genetic counseling can also be helpful for patients and their families. Rett syndrome treatment typically includes physical therapy, occupational therapy, speech and language therapy, nutritional support, and medications. Learn more about the Rett syndrome treatment @ New Treatment for Rett Syndrome In March 2023, the US FDA approved Acadia Pharmaceuticals' DAYBUE (trofinetide) as the first treatment for Rett syndrome in both adults and children aged two years and older. Trofinetide is a synthetic analog of a naturally occurring tripeptide called glycine-proline-glutamate (GPE). While the precise therapeutic mechanism of trofinetide in Rett syndrome remains unclear, animal studies have shown that it can promote dendritic branching and enhance synaptic plasticity. The medication is administered either orally or through a gastrostomy tube, taken twice daily—morning and evening—with or without food. Other Rett syndrome treatments, such as bromocriptine and carbidopa-levodopa (dopamine agonists), have been explored to manage motor symptoms in Rett syndrome, though their effectiveness tends to be limited and short-lived. Some evidence suggests levocarnitine may offer benefits in certain cases. For patients with gastroesophageal reflux (GER), antireflux medications like metoclopramide may be helpful, and anti-epileptic drugs are often used to manage seizure-like episodes. While none of these treatments offer a cure, they can help alleviate symptoms such as seizures, breathing irregularities, abnormal heart rhythms, indigestion, and constipation. Find out more on FDA-approved Rett syndrome drugs @ Rett Syndrome Treatment Options The pipeline for Rett syndrome is continuously evolving with ongoing research and clinical trials focusing on innovative approaches to improve outcomes and reduce side effects. Current areas of interest in the pipeline include targeted therapies, gene therapies, and others. Thus, the pipeline for Rett syndrome looks promising, with a range of novel therapies such as NA-921 (Bionetide) (Biomed Industries), NGN-401 (NEUROGENE), TSHA-102 (Taysha Gene Therapies), ANAVEX2-73(BLARCAMESINE) (Anavex Lifesciences), and others, and approaches under investigation that have the potential to redefine Rett syndrome treatment standards and improve patient outcomes in the future. Discover which therapies are expected to grab major Rett syndrome market share @ Rett Syndrome Market Report ANAVEX2-73 (blarcamesine), developed by Anavex Life Sciences, is an experimental oral treatment aimed at restoring normal brain signaling in individuals with Rett syndrome by activating the sigma-1 receptor (SIGMAR1), a key brain protein. The drug is currently being assessed in two clinical trials: The AVATAR study, a Phase III double-blind, randomized, placebo-controlled trial in adults (18+) with Rett syndrome. This study evaluates the drug's safety, tolerability, and efficacy using a range of clinical, molecular, and biochemical endpoints. The EXCELLENCE study, which is investigating its effects in pediatric patients aged 5–17 years. Results from an earlier Phase II trial in adult patients indicated behavioral improvements that persisted for several months during an extension phase. ANAVEX2-73 has received Fast Track, Rare Pediatric Disease, and Orphan Drug designations from both the FDA and the European Union. NGN-401, being developed by Neurogene, is an investigational one-time gene therapy for Rett syndrome based on an AAV9 vector. It is the first clinical candidate to deliver the full-length human MECP2 gene using Neurogene's proprietary EXACT™ technology, which precisely controls gene expression levels. This regulation is critical for treating Rett syndrome, as excessive MECP2 expression can lead to toxicity, a challenge for traditional gene therapies. Neurogene is currently conducting a Phase I/II clinical trial for NGN-401. In April 2025, the company published peer-reviewed research in Science Translational Medicine showcasing the ability of EXACT to maintain MECP2 expression at safe and therapeutic levels in preclinical Rett models. These findings supported the launch of their ongoing clinical trial. NGN-401 has received Fast Track, Rare Pediatric Disease, and Orphan Drug status from the FDA, along with Orphan and Advanced Therapy Medicinal Product designations from the EMA and Innovative Licensing and Access Pathway (ILAP) designation from the UK's MHRA. TSHA-102 is an experimental gene therapy delivered intrathecally using a self-complementary AAV9 vector, currently being studied for the treatment of Rett syndrome. Intended as a one-time therapy, it targets the underlying genetic cause of the disorder by introducing a functional MECP2 gene into cells of the central nervous system (CNS). The therapy features an innovative miRNA-Responsive Auto-Regulatory Element (miRARE) technology, which helps regulate MECP2 expression in individual CNS cells, minimizing the risk of overexpression. TSHA-102 has been granted multiple regulatory designations, including Regenerative Medicine Advanced Therapy, Fast Track, Orphan Drug, and Rare Pediatric Disease designations from the FDA, Orphan Drug designation from the European Commission, and an Innovative Licensing and Access Pathway designation from the UK's Medicines and Healthcare products Regulatory Agency. NA-921 (Bionetide) is an orally administered small molecule currently in Phase III clinical trials for treating Rett syndrome. Its ability to efficiently cross the blood-brain barrier allows it to be effective at low doses. In March 2025, Biomed Industries announced the results of its Phase II/III clinical trials. The topline data confirmed strong evidence of both safety and efficacy. A comparative analysis of side effects revealed that NA-921 has a significantly better safety and tolerability profile than DAYBUE, a drug marketed by Acadia Pharmaceuticals, Inc. These results position NA-921 as a promising new treatment option, with reduced side effects and higher patient adherence. Discover more about drugs for Rett syndrome in development @ Rett Syndrome Clinical Trials The anticipated launch of these emerging therapies for Rett syndrome are poised to transform the market landscape in the coming years. As these cutting-edge therapies continue to mature and gain regulatory approval, they are expected to reshape the Rett syndrome market landscape, offering new standards of care and unlocking opportunities for medical innovation and economic growth. DelveInsight estimates that the market size for Rett syndrome is expected to grow at a significant CAGR by 2034. The Rett syndrome market is estimated to change significantly for the forecast period 2025–2034. Major Rett syndrome market drivers, such as rising prevalence, technological advancements, and upcoming therapies, will boost the Rett syndrome market significantly. DelveInsight's latest published market report, titled as Rett Syndrome Market Insight, Epidemiology, and Market Forecast – 2034, will help you to discover which market leader is going to capture the largest market share. The report provides comprehensive insights into the Rett syndrome country-specific treatment guidelines, patient pool analysis, and epidemiology forecast to help understand the key opportunities and assess the market's underlying potential. The Rett syndrome market report proffers epidemiological analysis for the study period 2020–2034 in the 7MM segmented into: Total Rett Syndrome Diagnosed Prevalent Cases Rett Syndrome Gender-Specific Diagnosed Prevalent Cases Rett Syndrome Age-specific Diagnosed Prevalent Cases Rett Syndrome Treated Cases The report provides an edge while developing business strategies by understanding trends shaping and driving the 7MM Rett syndrome market. Highlights include: 10-year Forecast 7MM Analysis Epidemiology-based Market Forecasting Historical and Forecasted Market Analysis upto 2034 Emerging Drug Market Uptake Peak Sales Analysis Key Cross Competition Analysis Industry Expert's Opinion Access and Reimbursement Download this Rett syndrome market report to assess the epidemiology forecasts, understand the patient journeys, know KOLs' opinions about the upcoming treatment paradigms, and determine the factors contributing to the shift in the Rett syndrome market. Also, stay abreast of the mitigating factors to improve your market position in the Rett syndrome therapeutic space. Related Reports Rett Syndrome Epidemiology Forecast Rett Syndrome Epidemiology Forecast – 2034 report delivers an in-depth understanding of the disease, historical and forecasted Rett syndrome epidemiology in the 7MM, i.e., the United States, EU5 (Germany, Spain, Italy, France, and the United Kingdom), and Japan. Rett Syndrome Pipeline Rett Syndrome Pipeline Insight – 2025 report provides comprehensive insights about the pipeline landscape, pipeline drug profiles, including clinical and non-clinical stage products, and the key Rett syndrome companies, including Anavex Life Sciences Corp, Neurogene, Taysha GTx, among others. Angelman Syndrome Market Angelman Syndrome Market Insights, Epidemiology, and Market Forecast – 2034 report delivers an in-depth understanding of the disease, historical and forecasted epidemiology, as well as the market trends, market drivers, market barriers, and key Angelman syndrome companies, including GeneTx Biotherapeutics, GEXVal, Ionis Pharmaceuticals, Neuren Pharmaceuticals, Ovid Therapeutics, PTC Therapeutics, Roche, Sarepta Therapeutics, StrideBio, Taysha Gene Therapies, Ultragenyx Pharmaceutical, among others. Angelman Syndrome Pipeline Angelman Syndrome Pipeline Insight – 2025 report provides comprehensive insights about the pipeline landscape, pipeline drug profiles, including clinical and non-clinical stage products, and the key Angelman syndrome companies, including Neuren Pharmaceuticals, Ultragenyx Pharmaceutical, Ionis Pharmaceuticals, Roche, GEXVal, PTC Therapeutics, Taysha Gene Therapies, Ovid Therapeutics, among others. About DelveInsight DelveInsight is a leading Business Consultant and Market Research firm focused exclusively on life sciences. It supports pharma companies by providing comprehensive end-to-end solutions to improve their performance. Get hassle-free access to all the healthcare and pharma market research reports through our subscription-based platform PharmDelve. Contact Us Shruti Thakur info@ +14699457679 Logo: View original content:
Business Insider
14-05-2025
- Business
- Business Insider
UBS Sticks to Its Hold Rating for Great Portland Estates plc R.E.I.T. (GPE)
UBS analyst Zachary Gauge maintained a Hold rating on Great Portland Estates plc R.E.I.T. (GPE – Research Report) on May 12 and set a price target of p330.00. The company's shares closed yesterday at p321.50. Protect Your Portfolio Against Market Uncertainty Discover companies with rock-solid fundamentals in TipRanks' Smart Value Newsletter. Receive undervalued stocks, resilient to market uncertainty, delivered straight to your inbox. According to TipRanks, Gauge is ranked #4754 out of 9511 analysts. The word on The Street in general, suggests a Hold analyst consensus rating for Great Portland Estates plc R.E.I.T. with a p318.14 average price target. GPE market cap is currently £1.32B and has a P/E ratio of -33.63.
Associated Press
26-03-2025
- General
- Associated Press
In War-Torn Yemen, Education is Children's Only Hope
TAIZ, Yemen, March 26, 2025 (GLOBE NEWSWIRE) -- 10 years into the conflict, Yemen remains one of the largest humanitarian crises in the world, with children paying the highest price. Education is their only hope of building a better future. As the world marks the 10 year anniversary of the conflict, GPE and its partners re-affirm their commitment to Yemen's children, through continued initiatives to rehabilitate schools, train teachers, and expand psychosocial support in the face of ongoing crisis. Throughout the conflict, GPE has helped keep schools in Yemen open and safe for children to continue their learning. Working closely with the government, civil society and international partners, GPE has mobilized resources to sustain access to education and prevent the education system from collapsing. Yemen's decade-long conflict has left more than 4.5 million children out of school and disrupted the education of many more. Education is often the first service to be suspended and the last to be restored during conflict, with dire consequences for children and their wellbeing. Children's access to education during crises helps instill a sense of normalcy, fosters psychological resilience and supports long-term recovery and peacebuilding. Without education, a generation of Yemeni children are at risk of growing up without the skills they need to build their future and contribute to their countries' stability and prosperity. Attacks on students, teachers and schools have had a devastating impact. Around one in four schools in Yemen is unfit for use because it has been destroyed, damaged, turned into a shelter for the displaced or used for military purposes. 'I will not stop teaching. It is my duty to carry on what those before me did in teaching, so that education does not deteriorate further. Despite the difficult circumstances, I want children to get knowledge and education so they have better opportunities in the future,' - Arwa Hael, teacher, Saeed Hassan Fare'a school, Taiz, Yemen. The conflict has left Yemen's education system on the edge of collapse. GPE has worked with the government, civil society and other partners, including Save the Children, UNICEF, UNESCO, the World Bank and the World Food Programme, to avert a complete shutdown of the education system. Responding flexibly to the evolving needs of Yemen's education sector, this partnership has been able to provide critical support to children, including through rehabilitating damaged schools so children can resume their learning. Students have received basic school supplies and healthy meals. School meals have been critical, as the country is facing one of the worst hunger crises in the world, with more than half of the population struggling to access food. Approximately, 2.7 million children are suffering from acute malnutrition, including hundreds of thousands suffering from severe acute malnutrition – a life-threatening condition. Students have also received psychosocial support to help them cope with war-related trauma, including displacement, violence, military recruitment and lack of essential services. 'There was bombing one day, and my mom was scared and told me not to go to school. My first school was in the city of Taiz, and there was shelling nearby. There were fears, we did not feel safe, so we moved here where we feel safe. I started here in the first year of secondary school. If I don't get an education, I will be illiterate and ignorant. Education is the most important thing,' - Ragda Al-A'ameri, 17-year-old student, Saeed Hassan Fare'a school, Taiz, Yemen. More likely to be out of school, girls have received extra support to access learning. To address parents' security concerns about sending their daughters to school, over 2,000 female teachers have been hired to work in remote areas, encouraging more girls to go to school. Yemen is one of the first countries to join GPE in 2003 and has received GPE grants worth US$178 million. Throughout the conflict, over $125 million of this support has helped keep schools open across the most conflict-affected governorates and ensure students learn in a safe environment. GPE has brought together national and global actors and mobilized resources that have been critical in preventing the collapse of the education system. E: P: +33 7 82 26 07 18 P: +33 7 82 26 07 18
Associated Press
28-01-2025
- Health
- Associated Press
Education to Heal Ukrainian Children's Invisible War Scars
GPE is helping increase the capacity of Ukraine's educators to reinforce children's psychosocial resilience. More than 60,000 education professionals, including teachers, principals and school psychologists, are trained in psychosocial support and school safety. WASHINGTON, Jan. 28, 2025 (GLOBE NEWSWIRE) -- Since 2022, the war in Ukraine has eroded the mental health and well-being of the population, including approximately 5 million school-age children. ' There is no single child that is not affected by the war….61% of parents have reported to us that they have major concerns about their children's mental health and well-being because they are going through trauma. Even one single air alert during the day is enough to trigger anxiety and issues. ' - Munir Mammadzade, UNICEF Representative to Ukraine Displacement, violence and disruption of essential services has led to acute and chronic stress, anxiety, sadness, hopelessness, sleep problems, fatigue, irritability or anger among children. ' If children's mental health issues are not addressed, they cannot learn and develop to their full potential.' Danijel Cuturic, Education Programs Coordinator, UNESCO Ukraine Ensuring that children have access to education during crisis provides a sense of normalcy and social support through communication with peers and educators. However, according to the Ukrainian government, around 2,000 schools remain damaged or destroyed since the Russian invasion. Approximately 1.2 million students are accessing education online or through a blended format of online and in-person learning, relying on technology to stay connected with peers and educators. 'During the whole occupation, I have dreamed of studying because I was tired of just sitting at home and doing nothing. I did a little bit of research on the Internet, saw the subjects, tried to understand them by myself, but self-study was quite difficult. The most difficult thing was to keep your spirits up, because it was psychologically pressuring, very pressuring.' - Ivan Vasiliev, grade 11 student, Snihurivka Lyceum, Mykolaiv region Recognizing the critical need to support children through this difficult time, Ukraine has prioritized mental health within the education sector. As part of the National Program for Mental Health and Psychosocial Support led by Olena Zelenska, Ukraine's First Lady, the Ministry of Education and Science of Ukraine is working with partners to develop policies, protocols and tools to provide students with the mental support they need. One intervention includes training teams made up of school principals, teachers, school psychologists, security professionals, parents and caregivers across eight war-affected regions on school safety, psychosocial support and socioemotional learning. 'I have children in my class whose parents are soldiers. Some are waiting for their father or mother to call, some are waiting for both parents to call. These children need a lot of attention, support and help from teachers. We are an authority in the eyes of children, and we must be able to provide psychological help to them.' - Alyona Kolisnyk, primary school teacher, Balta Lyceum No. 2, Odesa region 'Before the war, we thought that we needed at least one professional in each school, like a psychologist. Right now, I think that all the teachers in Ukraine should have some level of experience in mental health support for their children because there are so many of them who need support from their teachers.' - Dmytro Zavgorodnii, Ukraine's Deputy Minister of Education and Science The training is one of several interventions that was made possible thanks to a GPE Multiplier grant of US$25.5 million, which matched a total of over $25 million in cash and in-kind from Microsoft, Google and UNESCO, totaling more than $51 million in education assistance for Ukraine. The funding helps the Ministry of Education and Science provide distance learning, psychosocial support, catch-up education and other activities that are being implemented by UNESCO and UNICEF for children in conflict-affected areas. A new GPE grant of $2.7 million, managed by the World Bank, aims to strengthen the Ministry of Education and Science's capacity to address the impact of war on students and their learning, support sector planning and implement reforms. About GPE GPE is the largest global fund solely dedicated to transforming education in lower-income countries, and a unique, multi-stakeholder partnership. We work to deliver quality education so that every girl and boy can have hope, opportunity and agency. For two decades, GPE has been delivering funds and supporting solutions to build strong and resilient education systems in countries characterized by extreme poverty or conflict so that more children, especially girls, get the education they need to thrive and contribute to building a more prosperous and sustainable world. GPE brings together all partners invested in education --lower-income countries, donors, international organizations, civil society, including youth and teacher organizations, the private sector and private foundations-- to transform education systems focusing particularly on the places and people with the greatest needs. Our unique model has helped partner countries make significant progress in improving access, learning and equity, and achieve better outcomes for hundreds of millions of boys and girls. P: +1 202 948 53 95
