Current clinical applications of in vivo gene therapy with AAVs
Hereditary diseases are caused by mutations in genes, and more than 7,000 rare diseases
affect over 30 million Americans. For more than 30 years, hundreds of researchers have …
affect over 30 million Americans. For more than 30 years, hundreds of researchers have …
Spinal muscular atrophy
Spinal muscular atrophy (SMA) is a neurodegenerative disorder caused by mutations in
SMN1 (encoding survival motor neuron protein (SMN)). Reduced expression of SMN leads …
SMN1 (encoding survival motor neuron protein (SMN)). Reduced expression of SMN leads …
Inflammation in CNS neurodegenerative diseases
J Stephenson, E Nutma, P van der Valk, S Amor - Immunology, 2018 - Wiley Online Library
Neurodegenerative diseases, the leading cause of morbidity and disability, are gaining
increased attention as they impose a considerable socioeconomic impact, due in part to the …
increased attention as they impose a considerable socioeconomic impact, due in part to the …
Five-year extension results of the phase 1 START trial of onasemnogene abeparvovec in spinal muscular atrophy
JR Mendell, SA Al-Zaidy, KJ Lehman, M McColly… - JAMA …, 2021 - jamanetwork.com
Importance This ongoing study assesses long-term safety and durability of response in
infants with spinal muscular atrophy (SMA) type 1 after dosing with onasemnogene …
infants with spinal muscular atrophy (SMA) type 1 after dosing with onasemnogene …
Onasemnogene abeparvovec: first global approval
SM Hoy - Drugs, 2019 - Springer
Onasemnogene abeparvovec (onasemnogene abeparvovec-xioi; formerly AVXS-101;
ZOLGENSMA®) is an adeno-associated viral vector-based gene therapy designed to deliver …
ZOLGENSMA®) is an adeno-associated viral vector-based gene therapy designed to deliver …
Correlation between SMA type and SMN2 copy number revisited: an analysis of 625 unrelated Spanish patients and a compilation of 2834 reported cases
M Calucho, S Bernal, L Alías, F March… - Neuromuscular …, 2018 - Elsevier
Spinal muscular atrophy (SMA) is a neuromuscular disorder caused by loss or mutations in
SMN1. According to age of onset, achieved motor abilities, and life span, SMA patients are …
SMN1. According to age of onset, achieved motor abilities, and life span, SMA patients are …
Newborn screening programs for spinal muscular atrophy worldwide: Where we stand and where to go
Spinal muscular atrophy (SMA) is a rare and devastating disease. New disease-modifying
treatments have recently been approved and early treatment has been related to a better …
treatments have recently been approved and early treatment has been related to a better …
Advances in treatment of spinal muscular atrophy–new phenotypes, new challenges, new implications for care
DC Schorling, A Pechmann… - Journal of …, 2020 - content.iospress.com
Abstract Spinal Muscular Atrophy (SMA) is caused by autosomal recessive mutations in
SMN1 and results in the loss of motor neurons and progressive muscle weakness. The …
SMN1 and results in the loss of motor neurons and progressive muscle weakness. The …
New treatments in spinal muscular atrophy: positive results and new challenges
S Messina, M Sframeli - Journal of clinical medicine, 2020 - mdpi.com
Spinal muscular atrophy (SMA) is one of the most common autosomal recessive diseases
with progressive weakness of skeletal and respiratory muscles, leading to significant …
with progressive weakness of skeletal and respiratory muscles, leading to significant …
[HTML][HTML] Therapeutic promise of carotenoids as antioxidants and anti-inflammatory agents in neurodegenerative disorders
Abstract Neurodegenerative disorders (NDs) including Alzheimer's disease, Parkinson's
disease, amyotrophic lateral sclerosis, Huntington's disease, and multiple sclerosis have …
disease, amyotrophic lateral sclerosis, Huntington's disease, and multiple sclerosis have …