https://www.medicalnewstoday.com/articles/gene-therapy-spinal-muscular-atrophy
Gene therapy for spinal muscular atrophy (SMA) has the potential to stop the progression of this condition. It works by replacing the damaged SMN1 gene that causes SMA with a functional one

https://www.youtube.com/watch?v=iBmyXr_o1hU
Connect with a specialist: http://bit.ly/2nIxt7tMeet Jerry Mendell, MD: http://bit.ly/2nCirzTMore about SMA Type 1: http://bit.ly/2nF1aWMMore on Research: ht

https://www.nejm.org/doi/full/10.1056/NEJMoa1706198
Spinal muscular atrophy (SMA) is a severe childhood monogenic disease resulting from loss or dysfunction of the gene encoding survival motor neuron 1 ( SMN1 ). The incidence of this disease is

https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10104684/
A study on assessing the impact of age and motor function on SMA Type 1 infants who received a single dose of gene replacement therapy (The recommended dose of Zolgensma is 1.1 × 1014 vector genomes per kilogram (vg/kg) of body weight) demonstrated that the early dosing and low motor group, with a mean age of 17 months, had a mean gain of 35.0

https://www.zolgensma.com/how-zolgensma-works
ZOLGENSMA ® (onasemnogene abeparvovec-xioi) is the only treatment that addresses the genetic cause of spinal muscular atrophy (SMA) with just one dose, and is a type of medicine called gene therapy.. Children with SMA are born with a missing or nonworking SMN1 gene. In a single infusion, ZOLGENSMA replaces the function of the SMN1 gene, by delivering a new, working SMN gene to the motor

https://www.mysmateam.com/resources/understanding-how-sma-gene-therapy-works
How Does Spinal Muscular Atrophy Gene Therapy Work? SMA gene therapy targets one of the major genes associated with the disease, notably SMN1 and SMN2. The U.S. Food and Drug Administration (FDA) ... Correlation Between SMA Type and SMN2 Copy Number Revisited: An Analysis of 625 Unrelated Spanish Patients and a Compilation of 2834 Reported

https://www.chop.edu/treatments/gene-therapy-spinal-muscular-atrophy-sma
Contact Us. 215-590-1719. Fax. 215-590-2223. Gene replacement therapy for spinal muscular atrophy (SMA) is offered as a treatment option for children who meet certain criteria. Gene replacement therapy for SMA is called onasemnogene abeparvovec-xioi (brand name Zolgensma).

https://www.healthline.com/health/spinal-muscular-atrophy/gene-therapy-for-spinal-muscular-atrophy
The gene therapy drug onasemnogene abeparvovec-xioi (Zolgensma) replaces the missing or nonworking SMN1 gene involved in SMA type 1, which can improve life expectancy and motor function. Spinal

https://www.zolgensma-hcp.com/about-zolgensma/moa/
ZOLGENSMA is a gene therapy designed to treat the genetic root cause of SMA 1. Spinal muscular atrophy (SMA) is caused by the deletion or mutation of the survival motor neuron 1 (SMN1) gene.The SMN1 gene produces survival motor neuron (SMN) protein that is critical for normal function of motor neurons.Patients with SMA have an insufficient amount of SMN protein, which leads to permanent loss

https://www.fda.gov/news-events/press-announcements/fda-approves-innovative-gene-therapy-treat-pediatric-patients-spinal-muscular-atrophy-rare-disease
The product is an adeno-associated virus vector-based gene therapy that targets the cause of SMA. The vector delivers a fully functional copy of human SMN gene into the target motor neuron cells.

https://www.nature.com/articles/gt201745
Spinal muscular atrophy (SMA) is a monogenic autosomal recessive disorder having an incidence of ~1 in 10 000 live births. 1, 2 Since the disease-causing genetic defect responsible for SMA was

https://www.nature.com/articles/s41434-022-00349-y
The EMA approved onasemnogene in March 2020 for the treatment of SMA patients with a biallelic mutation in the SMN1 gene and a clinical diagnosis for SMA type 1, or biallelic mutation in the SMN1

https://www.jmcp.org/doi/10.18553/jmcp.2018.24.12-a.s3
Spinal muscular atrophy (SMA) is an autosomal recessive neurodegenerative disease that, in most cases, involves deletion of a gene, SMN1, leading to a deficiency in survival motor neuron (SMN) protein.Although rare—occurring in 1 in 11,000 births 1 —it is nevertheless the most common fatal genetic disease of infancy. 2 Advancements in clinical care, especially technological improvements in

https://pubmed.ncbi.nlm.nih.gov/37065340/
The objective of the study is to review the safety and efficacy of a novel gene therapy, onasemnogene abeparvovec (Zolgensma), for SMA and assess current challenges for gene therapy. For this, we have conducted a literature search on PubMed, MEDLINE, and Ovid (2019 to 2022) in the English language using the terms SMA, onasemnogene, and gene

https://www.neurologylive.com/view/highly-effective-gene-therapies-sma-where-do-we-go-from-here
Current Treatment Options. There are 3 treatment options that target the root cause of SMA by increasing the amount of SMN protein ( TABLE ). Nusinersen (Spinraza; Biogen) and risdiplam (Evrysdi; Genentech) work by altering the splicing of SMN2 to increase the amount of functional SMN protein.

https://www.curesma.org/zolgensma/
Zolgensma ® (onasemnogene abeparvovec-xioi), marketed by Novartis Gene Therapies, is FDA-approved for patients with all forms and types of SMA who are under two years of age at the time of dosing; Zolgensma ® is given through an intravenous (IV) infusion that takes one hour. It is a one-time treatment; Zolgensma ® is an SMN-enhancing therapy that works by replacing the function of the

https://smauk.org.uk/treatments-research/zolgensma/zolgensma-what-how-faqs/
ZOLGENSMA™ is the trademark name for Onasemnogene abeparvovec, which is manufactured by the pharmaceutical company Novartis Gene Therapies. Zolgensma is a gene therapy that delivers a healthy copy of the SMN1 gene to motor neurons. It is made from parts of a virus called AAV9 (adeno-associated virus 9) that transports SMN1 around the body to many different cells, helping to restore some of

https://www.youtube.com/watch?v=yRrqbvUv6gQ
Connect with a specialist: http://bit.ly/2ZSoCz1How Gene Therapy works: http://bit.ly/2VpxIjaMore about Jerry Mendell, MD: http://bit.ly/2ZJEp2SMeet Jerry Me

https://www.semanticscholar.org/paper/Gene-replacement-therapy-for-spinal-muscular-safety-Mendon%C3%A7a-Ortega/82308f745312477bc188da84896673743e2efe4e
Onasemnogene abeparvovec is the first gene replacement therapy (GT) approved to treat this condition. An observational retrospective study was conducted to assess adverse events and efficacy of GT in SMA patients. Forty-one patients with SMA (58.5% females and 80.1% SMA type 1) were included. The mean age at GT dosing was 18 (±6.4) months.

https://www.evelinalondon.nhs.uk/resources/patient-information/zolgensma-SMA.pdf
a gene therapy that has been tested in clinical trials. It has been approved by th. NHS to treat some babies and children with SMA type 1. It can also be used to treat babies who have faults. n the SMN1 gene that mean they will develop SMA type 1. olgensma replaces the faulty SMN1 gene with a new copy. This can stop progression of SMA type 1

https://smanewstoday.com/news/preventive-dual-therapy-provides-modest-benefit-sma-type-1/
Three disease-modifying therapies are available for SMA. Evrysdi and Spinraza are given at regular intervals to boost SMN protein production by targeting the SMN2 gene, whereas Zolgensma is a one-time gene therapy that delivers a healthy copy of the SMN1 gene to motor neurons. "All three work best when given before the onset of weakness, but many preemptively treated children with two SMN2

https://smanewstoday.com/news/sma-gene-therapy-candidate-helps-young-children-sit-up-trial/
Exegenesis now testing gene therapy in young children with SMA type 1. According to Wu, Exegenesis is looking into developing EXG001-307 as a treatment for children with other forms of SMA in addition to type 1. "We are also exploring an accelerated development path in the slightly older SMA type 2/3 patient population," Wu said.

https://smanewstoday.com/news/infants-with-sma-type-1-treated-with-zolgensma-continue-to-achieve-milestones-show-improvements-in-motor-function-phase-3-trial-shows/
STR1VE-EU was one of the Phase 3 trials that assessed the safety and efficacy of Zolgensma. This trial involved 33 infants with SMA type 1 who were younger than 6 months at the time of treatment. All of the infants involved in the study had at least one copy of the SMN2 gene, and carried mutations in both copies of the SMN1 gene.. At the start of the study, nearly all (93.9%) infants were able

https://www.nature.com/articles/s41583-024-00829-7
Gene therapy is emerging as a powerful tool to modulate abnormal gene expression, a hallmark of most CNS disorders. The transformative potentials of recently approved gene therapies for the

https://www.news-medical.net/news/20240625/Comparative-study-on-preventative-SMA-therapies-shows-mixed-outcomes.aspx
They were divided into three groups: babies with two copies of SMN2 (SMA type 1) who received only gene therapy, babies with two copies of SMN2 (SMA type 1) who received gene therapy plus

https://smanewstoday.com/news/next-generation-sma-gene-therapy-voyager-novartis-goal/
Several SMA therapies work to slow or stop SMA progression by increasing the production of SMN. These include Evrysdi (risdiplam) and Spinraza (nusinersen), treatments that correct alternative splicing, and the gene therapy Zolgensma that delivers a healthy SMN1 gene using an virus (AAV) as a transport vehicle.. Voyager's AAV-based capsids may be safer, more effective than current AAVs

https://www.statnews.com/2024/06/21/vertex-stem-cell-therapy-trial-type-1-diabetes/
O RLANDO — Twelve people with type 1 diabetes who received a therapy derived from stem cells were able to produce enough of their own insulin to maintain healthy blood glucose levels 90 days