EMA Panel Backs Zolgensma Gene Therapy for SMA EMA Panel Backs Zolgensma Gene Therapy for SMA

Zolgensma is a gene therapy that addresses the genetic root cause of spinal muscular atrophy by replacing the defective or missing SMN1 gene to halt disease progression.International Approvals
Source: Medscape Neurology and Neurosurgery Headlines - Category: Neurology Tags: Neurology & Neurosurgery News Alert Source Type: news

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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
Semin Neurol DOI: 10.1055/s-0040-1702940In the past decade, the number of genes linked to neuromuscular diseases of childhood has expanded dramatically, and this genetic information is forming the basis for gene-specific and even mutation-specific therapies. At the forefront of these advances are the two recently approved treatments for spinal muscular atrophy: one, an antisense oligonucleotide that modifies splicing of the SMN2 gene, and, the other, a gene therapy vector that delivers the SMN1 gene to motor neurons, both of which are allowing patients to acquire developmental milestones previously unseen in this fatal dis...
Source: Seminars in Neurology - Category: Neurology Authors: Tags: Review Article Source Type: research
Abstract Spinal muscular atrophy (SMA) is a severe, inherited disease characterized by the progressive degeneration and death of motor neurons of the anterior horns of the spinal cord, which results in muscular atrophy and weakness of variable severity. Its early-onset form is invariably fatal in early childhood, while milder forms lead to permanent disability, physical deformities and respiratory complications. Recently, two novel revolutionary therapies, antisense oligonucleotides and gene therapy, have been approved, and might prove successful in making long-term survival of these patients likely. In this persp...
Source: Neurobiology of Disease - Category: Neurology Authors: Tags: Neurobiol Dis Source Type: research
From the growing range of patient entities through to new possibilities stemming from technology and patient-generated data, pharma has more and more ways to innovate when it comes to collaborating with patients. 
  Now pharma needs to identify the ones that will move the dial commercially. “Clearly there is a shift from patient engagement as a nice-to-have, feel-good activity, to something which is more strategic,” says Vanessa Pott, Director, Patient Advocacy&Strategic Partnerships at Merck. “It’s really important for us to stay abreast of all the external trends.”  To ...
Source: EyeForPharma - Category: Pharmaceuticals Authors: Source Type: news
Swiss drugmaker Novartis on Friday won a key European recommendation for its gene therapy Zolgensma against spinal muscular atrophy (SMA), clearing a hurdle for $2.1 million per patient treatment for approval in Europe within months.
Source: Reuters: Health - Category: Consumer Health News Tags: healthNews Source Type: news
Abstract Objective: To review the efficacy and safety of onasemnogene abeparvovec-xioi (Zolgensma) in the treatment of spinal muscular atrophy (SMA). Data Sources: An English-language literature search of PubMed, MEDLINE, and Ovid (1946 to December 2019) was completed using the terms onasemnogene, AVXS-101, and spinal muscular atrophy. Manufacturer prescribing information, article bibliographies, and data from ClinicalTrials.gov were incorporated in the reviewed data. Study Selection/Data Extraction: All studies registered on ClinicalTrials.gov were incorporated in the reviewed data. Data Synthesis: Onasemnogene i...
Source: The Annals of Pharmacotherapy - Category: Drugs & Pharmacology Authors: Tags: Ann Pharmacother Source Type: research
The objectives of this review are to discuss the current understanding of SMARD1 through a summary of the presently known information regarding its clinical presentation and pathogenesis and to discuss emerging therapeutic approaches. Advances in clinical care management have significantly extended the lives of individuals affected by SMARD1 and research into the molecular mechanisms that lead to the disease has identified potential strategies for intervention that target the underlying causes of SMARD1. Gene therapy via gene replacement or gene correction provides the potential for transformative therapies to halt or poss...
Source: Cellular and Molecular Life Sciences : CMLS - Category: Cytology Authors: Tags: Cell Mol Life Sci Source Type: research
Authors: Park JE, Yun SA, Roh EY, Yoon JH, Shin S, Ki CS Abstract Spinal muscular atrophy (SMA) is an autosomal recessive disease characterized by progressive proximal muscle weakness and atrophy. Given the recent introduction of gene therapies, knowledge of the SMA carrier frequency in various populations has become important for developing screening programs for this disease. In total, 1,581 anonymous DNA samples from an umbilical cord blood bank were tested for SMN1 and SMN2 gene copies using a multiplex ligation-dependent probe amplification assay. Twenty-nine of the 1,581 newborns [1.83%; 95% confidence interv...
Source: Annals of Laboratory Medicine - Category: Laboratory Medicine Tags: Ann Lab Med Source Type: research
This article reviews recent developments in the field of antisense and gene therapies for hereditary muscle diseases. PMID: 32076754 [PubMed - as supplied by publisher]
Source: Der Nervenarzt - Category: Neurology Authors: Tags: Nervenarzt Source Type: research
AbstractIn amyotrophic lateral sclerosis (ALS) and spinal muscular atrophy (SMA), spinal and lower brainstem motor neurons degenerate, but some motor neuron subtypes are spared, including oculomotor neurons (OMNs). The mechanisms responsible for this selective degeneration are largely unknown, but the molecular signatures of resistant and vulnerable motor neurons are distinct and offer clues to neuronal resilience and susceptibility. Here, we demonstrate that healthy OMNs preferentially expressSynaptotagmin 13 (SYT13) compared to spinal motor neurons. In end-stage ALS patients,SYT13 is enriched in both OMNs and the remaini...
Source: Acta Neuropathologica - Category: Neurology Source Type: research
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