Latest news with #RettSyndrome
Yahoo
2 days ago
- Business
- Yahoo
Taysha Gene Therapies Announces Details for Oral Presentations at the 2025 IRSF Rett Syndrome Scientific Meeting Reviewing Recent Updates from the TSHA-102 Clinical Program
Recently disclosed clinical cohort data from high (1x1015 total vg) and low dose (5.7x1014 total vg) TSHA-102 from REVEAL adolescent/adult and pediatric Phase 1/2 trials Caregiver research regarding gain/regain of developmental milestones supporting alignment with FDA on primary endpoint in the pivotal Part B trial of TSHA-102 Previously disclosed preclinical data supporting broad biodistribution across brain and spinal cord regions following lumbar intrathecal delivery of AAV9 gene therapy vectors in non-human primates Symposium on Rett syndrome natural history data findings DALLAS, June 03, 2025 (GLOBE NEWSWIRE) -- Taysha Gene Therapies, Inc. (Nasdaq: TSHA) (Taysha or the Company), a clinical-stage biotechnology company focused on advancing adeno-associated virus (AAV)-based gene therapies for severe monogenic diseases of the central nervous system (CNS), today announced details for three oral presentations reviewing recent updates supporting its TSHA-102 program in clinical evaluation for Rett syndrome, and a Taysha-hosted symposium on the Company's analysis of the natural history study data at the 2025 International Rett Syndrome Foundation (IRSF) Rett Syndrome Scientific Meeting, taking place in Boston from June 9-11, 2025. The three presentations will also be presented during a poster session on Monday, June 9, 2025, from 5:00-7:00 PM EST. Additional details on the meeting can be found at IRSF's website. 'We are excited to present data supporting our TSHA-102 clinical program and its potential to improve function or enable achievement of developmental milestones that would significantly improve quality of life. Importantly, these findings have increased our conviction in the differentiated potential of TSHA-102 to address the high unmet needs for patients and families suffering from this devastating disease,' said Sukumar Nagendran, M.D., President and Head of Research & Development at Taysha. 'We appreciate IRSF and their ongoing partnership, as well as the individuals with Rett syndrome, their caregivers and the clinicians who contributed to the important research that has increased our understanding of the disease progression and supported our alignment with the Food and Drug Administration (FDA) on a potential path to registration.' 2025 IRSF Rett Syndrome Scientific Meeting Oral Presentation Details Title: REVEAL Adolescent/Adult and Pediatric Clinical Trial Update: Safety and Efficacy Data on TSHA-102 AAV9 Investigational Gene Therapy in Clinical Evaluation for Rett Syndrome Date/Time: Wednesday, June 11, 2025, 9:30 AM ESTSession: Industry UpdatesPresenters: Elsa Rossignol, M.D., FRCP, FAAP, Associate Professor in Neuroscience and Pediatrics at the Université de Montréal, Director of the Rett Multidisciplinary Clinic of the CHU Sainte-Justine and Principal Investigator of the REVEAL Adolescent/Adult and Pediatric trials Title: Every Gain, Expanding Possibilities: Caregiver Insights on Meaningful Improvement in Rett Syndrome Gene Therapy (GT)Date/Time: Tuesday, June 10, 2025, 12:20 PM ESTSession: Biomarkers and Outcome MeasuresPresenters: Jenny Downs, MSc Ph.D., Program Head, Development and Disability at The Kids Research Institute Australia Title: rAAV9 Vector Biodistribution in Brain and Spinal Cord via Lumbar Intrathecal Infusion in Nonnhuman Primates (NHP): Assessing the Administration Route Leveraged in TSHA-102 Rett Syndrome Clinical Trials Date/Time: Tuesday, June 10, 2025, 10:10 AM ESTSession: Model Systems Presenters: Fred Porter, Ph.D., Chief of Staff and Technical Operations Officer of Taysha Taysha-Hosted Symposium: Gain and Regain of Developmental Milestones: How Natural History Insights are Redefining the Therapeutic Development for Rett SyndromeDate/Time: Tuesday, June 10, 2025, 7:30 AM ESTPresenter: Jeffrey Neul, M.D., Ph.D., Director, Vanderbilt Kennedy Center, Annette Schaffer Eskind Chair, Professor at the Vanderbilt University Medical Center, who served as Administrative Head of the Rett Syndrome Natural History Study About TSHA-102TSHA-102 is a self-complementary intrathecally delivered AAV9 investigational gene transfer therapy in clinical evaluation for Rett syndrome. Designed as a one-time treatment, TSHA-102 aims to address the genetic root cause of the disease by delivering a functional form of MECP2 to cells in the CNS. TSHA-102 utilizes a novel miRNA-Responsive Auto-Regulatory Element (miRARE) technology designed to mediate levels of MECP2 in the CNS on a cell-by-cell basis without risk of overexpression. TSHA-102 has received Regenerative Medicine Advanced Therapy, Fast Track and Orphan Drug and Rare Pediatric Disease designations from the FDA, Orphan Drug designation from the European Commission and Innovative Licensing and Access Pathway designation from the Medicines and Healthcare products Regulatory Agency. About Rett SyndromeRett syndrome is a rare neurodevelopmental disorder caused by mutations in the X-linked MECP2 gene encoding methyl CpG-binding protein 2 (MeCP2), which is essential for regulating neuronal and synaptic function in the brain. The disorder is characterized by loss of communication and hand function, slowing and/or regression of development, motor and respiratory impairment, seizures, intellectual disabilities and shortened life expectancy. Rett syndrome progression is divided into four key stages, beginning with early onset stagnation at 6 to 18 months of age followed by rapid regression, plateau and late motor deterioration. Rett syndrome primarily occurs in females and is one of the most common genetic causes of severe intellectual disability. Currently, there are no approved disease-modifying therapies that treat the genetic root cause of the disease. Rett syndrome caused by a pathogenic/likely pathogenic MECP2 mutation is estimated to affect between 15,000 and 20,000 patients in the U.S., EU, and U.K. About Taysha Gene TherapiesTaysha Gene Therapies (Nasdaq: TSHA) is a clinical-stage biotechnology company focused on advancing adeno-associated virus (AAV)-based gene therapies for severe monogenic diseases of the central nervous system. Its lead clinical program TSHA-102 is in development for Rett syndrome, a rare neurodevelopmental disorder with no approved disease-modifying therapies that address the genetic root cause of the disease. With a singular focus on developing transformative medicines, Taysha aims to address severe unmet medical needs and dramatically improve the lives of patients and their caregivers. The Company's management team has proven experience in gene therapy development and commercialization. Taysha leverages this experience, its manufacturing process and a clinically and commercially proven AAV9 capsid in an effort to rapidly translate treatments from bench to bedside. For more information, please visit Forward-Looking StatementsThis press release contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995. Words such as 'anticipates,' 'believes,' 'expects,' 'intends,' 'projects,' 'plans,' and 'future' or similar expressions are intended to identify forward-looking statements. Forward-looking statements include statements concerning the potential of TSHA-102, including the reproducibility and durability of any favorable results initially seen in patients dosed to date in clinical trials, including with respect to functional milestones, and our other product candidates to positively impact quality of life and alter the course of disease in the patients we seek to treat, our research, development and regulatory plans for our product candidates, including the timing of initiating additional trials, reporting data from our clinical trials and making regulatory submissions, communications from the FDA on the regulatory pathway for TSHA-102, and the potential for these product candidates to receive regulatory approval from the FDA or equivalent foreign regulatory agencies. Forward-looking statements are based on management's current expectations and are subject to various risks and uncertainties that could cause actual results to differ materially and adversely from those expressed or implied by such forward-looking statements. Accordingly, these forward-looking statements do not constitute guarantees of future performance, and you are cautioned not to place undue reliance on these forward-looking statements. Risks regarding our business are described in detail in our Securities and Exchange Commission (SEC) filings, including in our Annual Report on Form 10-K for the full-year ended December 31, 2024, and our Quarterly Report on Form 10-Q for the quarter ended March 31, 2025, which are available on the SEC's website at Additional information will be made available in other filings that we make from time to time with the SEC. These forward-looking statements speak only as of the date hereof, and we disclaim any obligation to update these statements except as may be required by law. Company Contact:Hayleigh Collins Senior Director, Corporate Communications and Investor Relations Taysha Gene Therapies, Media Contact:Carolyn HawleyInizio in retrieving data Sign in to access your portfolio Error in retrieving data Error in retrieving data Error in retrieving data Error in retrieving data
Yahoo
2 days ago
- Business
- Yahoo
Taysha Gene Therapies Announces Details for Oral Presentations at the 2025 IRSF Rett Syndrome Scientific Meeting Reviewing Recent Updates from the TSHA-102 Clinical Program
Recently disclosed clinical cohort data from high (1x1015 total vg) and low dose (5.7x1014 total vg) TSHA-102 from REVEAL adolescent/adult and pediatric Phase 1/2 trials Caregiver research regarding gain/regain of developmental milestones supporting alignment with FDA on primary endpoint in the pivotal Part B trial of TSHA-102 Previously disclosed preclinical data supporting broad biodistribution across brain and spinal cord regions following lumbar intrathecal delivery of AAV9 gene therapy vectors in non-human primates Symposium on Rett syndrome natural history data findings DALLAS, June 03, 2025 (GLOBE NEWSWIRE) -- Taysha Gene Therapies, Inc. (Nasdaq: TSHA) (Taysha or the Company), a clinical-stage biotechnology company focused on advancing adeno-associated virus (AAV)-based gene therapies for severe monogenic diseases of the central nervous system (CNS), today announced details for three oral presentations reviewing recent updates supporting its TSHA-102 program in clinical evaluation for Rett syndrome, and a Taysha-hosted symposium on the Company's analysis of the natural history study data at the 2025 International Rett Syndrome Foundation (IRSF) Rett Syndrome Scientific Meeting, taking place in Boston from June 9-11, 2025. The three presentations will also be presented during a poster session on Monday, June 9, 2025, from 5:00-7:00 PM EST. Additional details on the meeting can be found at IRSF's website. 'We are excited to present data supporting our TSHA-102 clinical program and its potential to improve function or enable achievement of developmental milestones that would significantly improve quality of life. Importantly, these findings have increased our conviction in the differentiated potential of TSHA-102 to address the high unmet needs for patients and families suffering from this devastating disease,' said Sukumar Nagendran, M.D., President and Head of Research & Development at Taysha. 'We appreciate IRSF and their ongoing partnership, as well as the individuals with Rett syndrome, their caregivers and the clinicians who contributed to the important research that has increased our understanding of the disease progression and supported our alignment with the Food and Drug Administration (FDA) on a potential path to registration.' 2025 IRSF Rett Syndrome Scientific Meeting Oral Presentation Details Title: REVEAL Adolescent/Adult and Pediatric Clinical Trial Update: Safety and Efficacy Data on TSHA-102 AAV9 Investigational Gene Therapy in Clinical Evaluation for Rett Syndrome Date/Time: Wednesday, June 11, 2025, 9:30 AM ESTSession: Industry UpdatesPresenters: Elsa Rossignol, M.D., FRCP, FAAP, Associate Professor in Neuroscience and Pediatrics at the Université de Montréal, Director of the Rett Multidisciplinary Clinic of the CHU Sainte-Justine and Principal Investigator of the REVEAL Adolescent/Adult and Pediatric trials Title: Every Gain, Expanding Possibilities: Caregiver Insights on Meaningful Improvement in Rett Syndrome Gene Therapy (GT)Date/Time: Tuesday, June 10, 2025, 12:20 PM ESTSession: Biomarkers and Outcome MeasuresPresenters: Jenny Downs, MSc Ph.D., Program Head, Development and Disability at The Kids Research Institute Australia Title: rAAV9 Vector Biodistribution in Brain and Spinal Cord via Lumbar Intrathecal Infusion in Nonnhuman Primates (NHP): Assessing the Administration Route Leveraged in TSHA-102 Rett Syndrome Clinical Trials Date/Time: Tuesday, June 10, 2025, 10:10 AM ESTSession: Model Systems Presenters: Fred Porter, Ph.D., Chief of Staff and Technical Operations Officer of Taysha Taysha-Hosted Symposium: Gain and Regain of Developmental Milestones: How Natural History Insights are Redefining the Therapeutic Development for Rett SyndromeDate/Time: Tuesday, June 10, 2025, 7:30 AM ESTPresenter: Jeffrey Neul, M.D., Ph.D., Director, Vanderbilt Kennedy Center, Annette Schaffer Eskind Chair, Professor at the Vanderbilt University Medical Center, who served as Administrative Head of the Rett Syndrome Natural History Study About TSHA-102TSHA-102 is a self-complementary intrathecally delivered AAV9 investigational gene transfer therapy in clinical evaluation for Rett syndrome. Designed as a one-time treatment, TSHA-102 aims to address the genetic root cause of the disease by delivering a functional form of MECP2 to cells in the CNS. TSHA-102 utilizes a novel miRNA-Responsive Auto-Regulatory Element (miRARE) technology designed to mediate levels of MECP2 in the CNS on a cell-by-cell basis without risk of overexpression. TSHA-102 has received Regenerative Medicine Advanced Therapy, Fast Track and Orphan Drug and Rare Pediatric Disease designations from the FDA, Orphan Drug designation from the European Commission and Innovative Licensing and Access Pathway designation from the Medicines and Healthcare products Regulatory Agency. About Rett SyndromeRett syndrome is a rare neurodevelopmental disorder caused by mutations in the X-linked MECP2 gene encoding methyl CpG-binding protein 2 (MeCP2), which is essential for regulating neuronal and synaptic function in the brain. The disorder is characterized by loss of communication and hand function, slowing and/or regression of development, motor and respiratory impairment, seizures, intellectual disabilities and shortened life expectancy. Rett syndrome progression is divided into four key stages, beginning with early onset stagnation at 6 to 18 months of age followed by rapid regression, plateau and late motor deterioration. Rett syndrome primarily occurs in females and is one of the most common genetic causes of severe intellectual disability. Currently, there are no approved disease-modifying therapies that treat the genetic root cause of the disease. Rett syndrome caused by a pathogenic/likely pathogenic MECP2 mutation is estimated to affect between 15,000 and 20,000 patients in the U.S., EU, and U.K. About Taysha Gene TherapiesTaysha Gene Therapies (Nasdaq: TSHA) is a clinical-stage biotechnology company focused on advancing adeno-associated virus (AAV)-based gene therapies for severe monogenic diseases of the central nervous system. Its lead clinical program TSHA-102 is in development for Rett syndrome, a rare neurodevelopmental disorder with no approved disease-modifying therapies that address the genetic root cause of the disease. With a singular focus on developing transformative medicines, Taysha aims to address severe unmet medical needs and dramatically improve the lives of patients and their caregivers. The Company's management team has proven experience in gene therapy development and commercialization. Taysha leverages this experience, its manufacturing process and a clinically and commercially proven AAV9 capsid in an effort to rapidly translate treatments from bench to bedside. For more information, please visit Forward-Looking StatementsThis press release contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995. Words such as 'anticipates,' 'believes,' 'expects,' 'intends,' 'projects,' 'plans,' and 'future' or similar expressions are intended to identify forward-looking statements. Forward-looking statements include statements concerning the potential of TSHA-102, including the reproducibility and durability of any favorable results initially seen in patients dosed to date in clinical trials, including with respect to functional milestones, and our other product candidates to positively impact quality of life and alter the course of disease in the patients we seek to treat, our research, development and regulatory plans for our product candidates, including the timing of initiating additional trials, reporting data from our clinical trials and making regulatory submissions, communications from the FDA on the regulatory pathway for TSHA-102, and the potential for these product candidates to receive regulatory approval from the FDA or equivalent foreign regulatory agencies. Forward-looking statements are based on management's current expectations and are subject to various risks and uncertainties that could cause actual results to differ materially and adversely from those expressed or implied by such forward-looking statements. Accordingly, these forward-looking statements do not constitute guarantees of future performance, and you are cautioned not to place undue reliance on these forward-looking statements. Risks regarding our business are described in detail in our Securities and Exchange Commission (SEC) filings, including in our Annual Report on Form 10-K for the full-year ended December 31, 2024, and our Quarterly Report on Form 10-Q for the quarter ended March 31, 2025, which are available on the SEC's website at Additional information will be made available in other filings that we make from time to time with the SEC. These forward-looking statements speak only as of the date hereof, and we disclaim any obligation to update these statements except as may be required by law. Company Contact:Hayleigh Collins Senior Director, Corporate Communications and Investor Relations Taysha Gene Therapies, Media Contact:Carolyn HawleyInizio
Yahoo
26-05-2025
- Health
- Yahoo
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: SOURCE DelveInsight Business Research, LLP
Yahoo
24-05-2025
- Health
- Yahoo
The Story I Never Got To Report: A Medical Breakthrough That Could Have Saved My Son
Journalists, by profession and nature, look for stories. We search for what's new, important, long-awaited, surprising or hidden so we can share it and help people understand the world. There is no better job. We're also competitive, scanning headlines and bylines, looking at what others have found to see if we've been scooped. Last week I read a headline in the New York Times that stopped me in my tracks. It was the story I wished with all my soul I could have written myself a few years ago, before it was too late for my son Henry. I'd already written it in my head. And yet there it was in front of me. 'Baby Is Healed With World's First Personalized Gene-Editing Treatment.' The sub-headline declared: 'The technique used on a 9½-month-old boy with a rare condition has the potential to help people with thousands of other uncommon genetic diseases.' Henry, who passed away two years ago this summer, had one of those other uncommon genetic diseases. It is called Rett syndrome and is caused by a tiny mutation of a gene called MECP2 in our DNA. I didn't know what MECP2 was until the diagnosis. The names of genetic diseases are codes. More specifically, they are coordinates that locate on the genetic map the exact spot where an error is located. Henry's problem, the root of the issue that prevented him from walking, talking and breathing properly, was right there in the MECP2 gene. Yet it remained unreachable. Genetic diseases tease you. You stare into a glass box. You can see the culprit, but can not touch him. If only that little genetic quirk, that typo among billions of coded characters could be repaired, then everything else would fall into place. 'The baby, now 9 ½ months old,' The New York Times reported, 'became the first patient of any age to have a custom gene-editing treatment, according to his doctors. He received an infusion made just for him and designed to fix his precise mutation.' I wished I could have broken the story with Henry as patient zero. I had imagined the roll-out too, coming back on the set of TODAY with Henry and his mother Mary, who has written about Henry's life and losing him for We'd sit with Savanah Guthrie, who has been supporting ongoing research using Henry's cells, and talk — cautiously, hopefully and thankfully — about the progress we were seeing. I allowed myself to imagine saying that Henry was starting to speak. He had been awakened, cured and reborn. There wouldn't be a dry eye in the studio. I never got to do that story about Henry. Sometimes our timelines don't overlap with scientific progress. They rarely do. Mary and I are full of nothing but joy that from now on, so many other families will be able to write new and wonderful stories of their own. This article was originally published on
Yahoo
23-05-2025
- Entertainment
- Yahoo
NBC's Richard Engel Shares Heartbreaking Reaction to New Medical Breakthrough That Could Potentially Have Saved His Son
Richard Engel penned an emotional personal essay about the first-ever personalized gene-editing treatment, which potentially could have saved his son Henry's life The NBC News chief foreign correspondent announced with a post on X in August 2022 that he lost his son after a years-long battle with Rett Syndrome Engel is also a father to son Theodore, 5, whom he shares with his wife MaryRichard Engel candidly shared his emotions about a recent medical breakthrough. After reading a New York Times report about a baby being healed from a rare genetic disorder with the first-ever personalized gene-editing treatment, the NBC News chief foreign correspondent, 51, penned an emotional personal essay about how the new medical breakthrough could have potentially saved his son Henry's life, who died in 2022 after being diagnosed with Rett Syndrome. "I wished I could have broken the story with Henry as patient zero," he wrote in the essay published on TODAY. "I had imagined the roll-out too, coming back on the set of TODAY with Henry and his mother Mary, who has written about Henry's life and losing him for "We'd sit with Savanah Guthrie, who has been supporting ongoing research using Henry's cells, and talk — cautiously, hopefully and thankfully — about the progress we were seeing," continued Engel. "I allowed myself to imagine saying that Henry was starting to speak. He had been awakened, cured and reborn. There wouldn't be a dry eye in the studio." Never miss a story — sign up for to stay up-to-date on the best of what PEOPLE has to offer, from celebrity news to compelling human interest stories. "I never got to do that story about Henry. Sometimes our timelines don't overlap with scientific progress. They rarely do," he wrote. "Mary and I are full of nothing but joy that from now on, so many other families will be able to write new and wonderful stories of their own." The journalist first announced with a post on X in August 2022 that his 6-year-old son had died after a years-long battle with Rett Syndrome, a rare genetic neurological disorder that leads to severe physical and cognitive impairments and has no cure, according to the Mayo Clinic. Henry, whom Richard shares with his wife Mary, died on Aug. 9, according to a memorial page on the Texas Children's Hospital website. "Our beloved son Henry passed away. He had the softest blue eyes, an easy smile and a contagious giggle. We always surrounded him with love and he returned it, and so much more," Engel wrote, before giving another update in a follow-up post, "Researchers are making amazing progress using Henry's cells to help cure Rett Syndrome so others don't have to endure this terrible disease." Henry was first diagnosed with the disorder in 2017. Engel spoke to PEOPLE in January 2018 about his son's diagnosis, noting at the time that his son was "probably not going to walk, probably not going to speak, probably not going to have any mental capacity beyond the level of a 2-year-old." Months later, in October 2018, the journalist opened up in a candid essay for Today about his family's harrowing journey and his son's condition. At the time, Engel said it was getting "increasingly difficult" to help Henry keep active "because of basic physics," as the parents had to move their son constantly to help keep him engaged in his surroundings — something that led Engel's wife Mary to start "working out like mad" to keep up with the physical demands of parenting. is now available in the Apple App Store! Download it now for the most binge-worthy celeb content, exclusive video clips, astrology updates and more! In August 2019, Engel and his wife welcomed another little boy, son Theodore, into their family and spoke with PEOPLE about the adjustment at home. Engel said that it would be "difficult" to watch baby Theo pass their son Henry, 3 at the time, with certain developmental milestones. "We know why there is this differential. And it's going to be hard to watch Theo pass his older brother in terms of capabilities," he said. "That's going to be very difficult for us to see. To see a 1-month-old very soon overcoming his almost-4-year-old brother … that's going to be tough." Read the original article on People
