Newly Approved Spinal Muscular Atrophy Gene Therapy, Zolgensma ® ,...

AskBio continues to lead the path for gene therapy development and cost-effective manufacturing methods based on the contributions of its co-founder, Dr. Jude Samulski.(PRWeb May 30, 2019)Read the full story at https://www.prweb.com/releases/newly_approved_spinal_muscular_atrophy_gene_therapy_zolgensma_validates_askbio_gene_therapy_platform/prweb16344912.htm
Source: PRWeb: Medical Pharmaceuticals - Category: Pharmaceuticals Source Type: news

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Until recently, supportive care was the sole treatment option for patients with spinal muscular atrophy (SMA). Disease-modifying treatments (eg, gene therapy [onasemnogene abeparvovec, formerly AVXS-101], antisense oligonucleotides [nusinersen]) are now available and have dramatically improved prognosis. While, in the context of this rapidly evolving field, some patients have received more than one of the different disease-modifying treatments, real-world safety and efficacy data in in these patients remain limited.
Source: Neuromuscular Disorders - Category: Neurology Authors: Source Type: research
Spinal muscular atrophy (SMA) is caused by deletion/mutation of the survival motor neuron 1 gene (SMN1). AVXS-101 IT is a one-time gene therapy designed to deliver fully functional copy of human SMN gene to address the genetic root cause of SMA. STRONG (CL-102, NCT03381729), a phase 1, open-label study, assesses the safety, optimal dose, and efficacy of AVXS-101 IT. Patients (pts; biallelic SMN1 loss, 3xSMN2) aged ≥6
Source: Neuromuscular Disorders - Category: Neurology Authors: Source Type: research
In conclusion, this in-depth longitudinal transcriptomics analysis in SMA reveals novel cell type–specific changes that, alone and combined, present compelling targets, including Gdf15, for future gene therapy studies aimed toward preserving vulnerable motor neurons.
Source: Genome Research - Category: Genetics & Stem Cells Authors: Tags: RESEARCH Source Type: research
(Nationwide Children's Hospital) A new study confirms the safety and efficacy of gene therapy in children with spinal muscular atrophy under two years old.
Source: EurekAlert! - Biology - Category: Biology Source Type: news
on CL Abstract Spinal Muscular Atrophy (SMA) is an autosomal recessive neuromuscular disease caused by deletions or mutations in the survival motor neuron (SMN1) gene. An important hallmark of disease progression is the pathology of neuromuscular junctions (NMJs). Affected NMJs in the SMA context exhibit delayed maturation, impaired synaptic transmission, and loss of contact between motor neurons and skeletal muscle. Protection and maintenance of NMJs remains a focal point of therapeutic strategies to treat SMA, and the recent implication of the NMJ-organizer Agrin in SMA pathology suggests additional NMJ organizi...
Source: Biochemical and Biophysical Research communications - Category: Biochemistry Authors: Tags: Biochem Biophys Res Commun Source Type: research
In conclusion, this in-depth longitudinal transcriptomics analysis in SMA reveals novel cell type–specific changes that, alone and combined, present compelling targets, including Gdf15, for future gene therapy studies aimed toward preserving vulnerable motor neurons.
Source: Genome Research - Category: Genetics & Stem Cells Authors: Tags: RESEARCH Source Type: research
Abstract The technique of gene therapy, ever since its advent nearly fifty years ago, has been utilized by scientists as a po-tential treatment option for various disorders. This review discusses some of the major neurodegenerative diseases (NDDs) like Alzheimer's disease (AD), Parkinson's disease (PD), Motor neuron diseases (MND), Spinal muscular atrophy (SMA), Huntington's disease (HD), Multiple sclerosis (MS) etc. and their underlying genetic mechanisms along with therole that gene therapy can play in combating them. The pathogenesis and the molecular mechanisms specifying the altered gene ex-pression of each o...
Source: Current Gene Therapy - Category: Genetics & Stem Cells Authors: Tags: Curr Gene Ther Source Type: research
Contributors : Eva Hedlund ; Susanne Nichterwitz ; Helena Storvall ; Rickard SandbergSeries Type : Expression profiling by high throughput sequencingOrganism : Mus musculusMotor neurons are selectively vulnerable in spinal muscular atrophy (SMA). However, some brainstem motor neuron groups, including oculomotor and trochlear, which innervate the muscles around the eyes, are for unknown reasons spared. Here, we investigate the transcriptional dynamics in discrete neuronal populations in health and SMA to identify mechanisms of vulnerability and resistance. Such mechanisms could reveal targets for future gene therapy studies...
Source: GEO: Gene Expression Omnibus - Category: Genetics & Stem Cells Tags: Expression profiling by high throughput sequencing Mus musculus Source Type: research
Spinal muscular atrophy (SMA) is a common and lethal autosomal recessive neurodegenerative disease caused by mutations in the survival motor neuron 1 (SMN1) gene. At present, gene therapy medicine for SMA, i.e...
Source: BMC Medical Genetics - Category: Genetics & Stem Cells Authors: Tags: Research article Source Type: research
CONCLUSION: Gene replacement therapy with onasemnogene abeparvovec has the potential to significantly influence the course of SMA. Long-term data on sustainability of effects and possible adverse effects of gene replacement therapy are not yet available. The application of this innovative therapy must be carried out in specialized and appropriately qualified treatment centers under strict safety conditions. This article makes suggestions for the necessary framework conditions and gives recommendations for a systematic pretreatment and posttreatment assessment schedule under gene therapy. The effectiveness and safety o...
Source: Der Nervenarzt - Category: Neurology Authors: Tags: Nervenarzt Source Type: research
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