Jordana Holovach (12.22.2025) 4 minsVector gene therapy represents one of the most consequential advances in modern medicine, redefining how genetic diseases are treated at their root cause rather than managed symptomatically.
Read MoreJordana Holovach (10.28.2025) 4 minsIn the realm of rare diseases, Canavan Disease has continuously presented itself as a complex puzzle that researchers have been committed to deciphering.
Read MoreJordana Holovach (09.08.2025) 4 minsWhite matter is the brain’s vital communication tissue, made up of nerve fibers coated in myelin that help signals travel efficiently. Understanding how leukodystrophies, a group of rare neurological disorders, damage white matter is key to making sense of these complex neurological disorders.
Read MoreJordana Holovach (07.08.2025) 4 minsFew conditions are as quietly devastating as leukodystrophies, these rare neurological disorders relentlessly target the brain’s white matter.
Read MoreJordana Holovach (06.24.2025) 4 minsThe Canavan disease treatment market remains small and highly specialized, reflecting the ultra-rare nature of the disorder.
Read MoreJordana Holovach (04.24.2025) 4 minsDNA may shape white matter by influencing myelin maintenance, brain repair, and aging-related disease vulnerability.
Read MoreJordana Holovach (04.23.2025) 4 minsNeural pathways link thought and movement; white matter disease disrupts them, impairing physical and cognitive function.
Read MoreJordana Holovach (04.21.2025) 4 minsCanavan disease is a rare genetic disorder that primarily affects the brain, often presenting subtle signs in infancy that can be easy to overlook.
Read MoreJordana Holovach (04.10.2025) 4 minsCanavan disease is a rare genetic disorder that primarily affects the brain, often presenting subtle signs in infancy that can be easy to overlook.
Read MoreJordana Holovach (03.25.2025) 4 minsCanavan disease is a rare genetic disorder that primarily affects the brain, often presenting subtle signs in infancy that can be easy to overlook.
Read MoreJordana Holovach (03.13.2025) 4 minsCanavan disease treatment remains a significant hurdle, as this rare and fatal neurological disorder continues to impact both the medical community and affected families.
Read MoreJordana Holovach (03.05.2025) 4 minsCanavan disease treatment remains a significant hurdle, as this rare and fatal neurological disorder continues to impact both the medical community and affected families.
Read MoreJordana Holovach (02.13.2025) 4 minsThe silent disruptor, white matter disease, it impacts brain communication, motor skills, and cognitive health worldwide, demanding urgent research and innovative treatment advancements.
Read MoreJordana Holovach (01.22.2025) 4 minsStudies like the one on AAV/Olig001 are critical for advancing our understanding of gene therapy’s potential to treat debilitating white matter diseases such as Canavan disease. Research into targeted therapies offers hope for addressing the underlying causes of these conditions, rather than just managing symptoms.
Read MoreJordana Holovach (01.22.2025) 4 minsReceiving a diagnosis of Canavan disease for your child is a profoundly emotional and challenging experience. Canavan disease is a rare genetic disorder affecting the brain’s white matter, leading to developmental delays, motor skill challenges, and progressive neurological impairment.
Read MoreJordana Holovach (01.08.2025) 4 minsWhite matter diseases are a group of neurological disorders that often fly under the radar on the national stage but play a critical role in brain function.
Read MoreJordana Holovach (01.08.2025) 4 minsNavigating the complexities of Canavan disease inheritance can be both confusing and concerning for families. Comprehending the genetics of the disease can provide clarity and guide families in making informed decisions. Genetic insights not only shed light on how the condition is passed down but also offer hope for proactive planning.
Read MoreJordana Holovach (01.06.2025) 3 minsWhite matter diseases, a group of rare neurological disorders, are more common than many realize, affecting more than half of individuals aged 60 and older, as shown by brain scan studies.
Read MoreJordana Holovach (12.11.2024) 3 minsCanavan disease is a severe neuro-degenerative condition that negatively affects the white matter of the brain. Spotting this early in your child can improve the chances of mitigating the condition’s effects, unlocking timely interventions and hope for managing this journey, as outlined in this resource.
Read MoreJordana Holovach (12.09.2024) 3 minsThe rare disease landscape is vast and it encompasses a variety of symptoms. Some signs occur early and are more easily detectable, while other indicators are more stealthy.
Read MoreJordana Holovach (10.28.2024) 3 minsRare diseases each present their own unique signs, and recognizing these indicators is crucial for accurate diagnosis and effective treatment. A rare disease such as Canavan disease is a complex condition that affects the brain’s white matter during infancy. Identifying its indicators is crucial, especially given the time constraints for effective intervention.
Read MoreJordana Holovach (10.17.2024) 3 minsRare diseases each present their own unique signs, and recognizing these indicators is crucial for accurate diagnosis and effective treatment. A rare disease such as Canavan disease is a complex condition that affects the brain’s white matter during infancy. Identifying its indicators is crucial, especially given the time constraints for effective intervention.
Read MoreJordana Holovach (09.20.2024) 3 minsRare diseases each present their own unique signs, and recognizing these indicators is crucial for accurate diagnosis and effective treatment. A rare disease such as Canavan disease is a complex condition that affects the brain’s white matter during infancy. Identifying its indicators is crucial, especially given the time constraints for effective intervention.
Read MoreJordana Holovach (09.17.2024) 3 minsRare diseases each present their own unique signs, and recognizing these indicators is crucial for accurate diagnosis and effective treatment. A rare disease such as Canavan disease is a complex condition that affects the brain’s white matter during infancy. Identifying its indicators is crucial, especially given the time constraints for effective intervention.
Read MoreJordana Holovach (08.29.2024) 3 minsRare diseases each present their own unique signs, and recognizing these indicators is crucial for accurate diagnosis and effective treatment. A rare disease such as Canavan disease is a complex condition that affects the brain’s white matter during infancy. Identifying its indicators is crucial, especially given the time constraints for effective intervention.
Read MoreJordana Holovach (08.27.2024) 3 minsRare diseases each present their own unique signs, and recognizing these indicators is crucial for accurate diagnosis and effective treatment. A rare disease such as Canavan disease is a complex condition that affects the brain’s white matter during infancy. Identifying its indicators is crucial, especially given the time constraints for effective intervention.
Read MoreJordana Holovach (08.05.2024) 3 minsRare diseases each present their own unique signs, and recognizing these indicators is crucial for accurate diagnosis and effective treatment. A rare disease such as Canavan disease is a complex condition that affects the brain’s white matter during infancy. Identifying its indicators is crucial, especially given the time constraints for effective intervention.
Read MoreJordana Holovach (07.08.2024) 3 minsRare diseases each present their own unique signs, and recognizing these indicators is crucial for accurate diagnosis and effective treatment. A rare disease such as Canavan disease is a complex condition that affects the brain’s white matter during infancy. Identifying its indicators is crucial, especially given the time constraints for effective intervention.
Read MoreJordana Holovach (07.04.2024) 3 minsRare diseases each present their own unique signs, and recognizing these indicators is crucial for accurate diagnosis and effective treatment. A rare disease such as Canavan disease is a complex condition that affects the brain’s white matter during infancy. Identifying its indicators is crucial, especially given the time constraints for effective intervention.
Read MoreJordana Holovach (06.09.2024) 3 minsRecent decades have seen significant technological advancements that have propelled the treatment of rare diseases forward.
Read MoreJordana Holovach (06.05.2024) 3 minsCanavan disease is a rare, inherited neurological disorder that primarily affects the brain’s development and function.
Read MoreJordana Holovach (04.30.2024) 3 minsCanavan disease is a rare, inherited neurological disorder that primarily affects the brain’s development and function.
Read MoreJordana Holovach (04.23.2024) 3 minsCanavan disease is a rare, inherited neurological disorder that primarily affects the brain’s development and function.
Read MoreJordana Holovach (04.15.2024) 3 minsCanavan disease is a rare, inherited neurological disorder that primarily affects the brain’s development and function.
Read MoreJordana Holovach (04.11.2024) 3 minsCanavan disease is a complex ailment and a member of rare diseases discovered in the 20th century.
Read MoreJordana Holovach (04.09.2024) 3 minsRecent advancements in rare disease technology have catapulted gene therapy research to new heights.
Read MoreRick Layer (03.25.2024) 3 minsA Q&A with Rick Layer, Vice President, Myrtelle’s Head of Pharmacology and Translational Science
Read MoreJordana Holovach (10.18.2022) 3 minsCheryl Demos Fludas, Head of Clinical Operations at Myrtelle, explains the process of enrolling a patient in an experimental clinical trial, emphasizing the importance of informed consent and eligibility criteria.
Read MoreJordana Holovach (08.16.2022) 9 minsChristopher J. Morrison, Senior Vice President of Technical Operations, discusses the complexities of gene therapy manufacturing, the importance of cGMP standards, manufacturing timelines and challenges, yield determination, and the stringent measures taken to ensure the safety and quality of gene therapy products.
Read MoreJordana Holovach (06.07.2022) 3 minsIn this Q&A with Richard Layer, Vice President and Head of Pharmacology, key insights into the central function of oligodendrocytes and myelin generation, the role of Myrtelle’s novel class of nonpathogenic rAAV vectors in gene therapy for myelin-related disorders, and the expansion of Myrtelle’s pipeline to address Pelizaeus-Merzbacher Disease (PMD) are discussed. Additionally, the evolving understanding of oligodendrocytes’ importance in the central nervous system and their potential implications for various neurological and psychiatric disorders is highlighted.
Read MoreJordana Holovach (04.20.2022) 3 minsNancy Barone Kribbs, Senior Vice President of Global Regulatory Affairs at Myrtelle, discusses the regulatory path for Myrtelle’s rAAV-Olig001-ASPA gene therapy, emphasizing the importance of establishing efficacy and safety in drug development for treating Canavan disease patients.
Read MoreJordana Holovach (03.11.2022) 4 minsArmen’s passion is to bring novel transformational treatments to rare disease patients through innovative modalities.
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