nusinersen

01/01/2020 - "Elderly patient with 5q spinal muscular atrophy type 4 markedly improved by Nusinersen."
11/01/2020 - "Nusinersen has recently been approved and more widely used as first-line treatment of spinal muscular atrophy (SMA). "
01/01/2021 - "Our findings are consistent with other trials of nusinersen and show improved survival and attainment of motor milestones over 3 years in patients with infantile-onset spinal muscular atrophy, with a favourable safety profile. "
07/01/2023 - "Nusinersen was effective in improving motor function and survival in infantile and childhood-onset spinal muscular atrophy (SMA), and the value of real-world experiences in adult SMA patients increase gradually. "
07/01/2022 - "Safety, tolerability, and efficacy of a widely available nusinersen program for Polish children with Spinal Muscular Atrophy."

01/01/2021 - "Despite functional gain and motor stabilization, treatment with nusinersen did not prevent the progression of scoliosis.ConclusionsOur data provide evidence for the long-term safety and efficacy of nusinersen use in the treatment of later-onset SMA, and patients with shorter disease duration showed better response to treatment."
03/01/2023 - "The degree of muscle damage may confer the variability in response to nusinersen in SMA types 2 and 3. Muscle MRI score along with the severity of scoliosis assessed at baseline may help to predict the motor function change."
08/01/2022 - "The objective of this study is to document the development and progression of scoliosis in patients with SMA1 treated with nusinersen. "
01/01/2020 - "This is a retrospective, single-centre study investigating the feasibility and safety of CT-guided, transforaminal, lumbar puncture for the intrathecal administration of nusinersen (Spinranza®; Biogen; Cambridge, MA, USA) in a cohort of adult subjects with SMA type 2, severe neuromuscular scoliosis and previous spinal surgery. "
09/01/2023 - "It is feasible and suitable to carry out intrathecal nusinersen injection under ultrasound combined with three-dimensional CT imaging for children with severe scoliosis."

11/11/2021 - "Improvement in Fine Manual Dexterity in Children with Spinal Muscular Atrophy Type 2 after Nusinersen Injection: A Case Series."
08/01/2021 - "Nusinersen improved respiratory function in spinal muscular atrophy type 2."
09/01/2020 - "Two Years of Improved Neurological Function With Nusinersen in a 48-Year-Old Patient With Spinal Muscular Atrophy Type 3."
01/01/2021 - "Based on the results of this study, we suggested that nusinersen is well-tolerated and efficacious when treating older children and adult patients with spinal muscular atrophy 1, 2, and 3."
01/01/2020 - "Nusinersen treatment and cerebrospinal fluid neurofilaments: An explorative study on Spinal Muscular Atrophy type 3 patients."

01/01/2022 - "Previous papers on ultrasound (US)-guided delivery of nusinersen in patients with spinal muscle atrophy (SMA) described good results with translaminar approach. "
03/01/2021 - "Efficacy of nusinersen in adult 5q-spinal muscular atrophy (SMA) patients regarding motor function has recently been demonstrated. "
01/01/2023 - "Nusinersen, an extremely expensive biologic drug (around 100,000 US$ per dose) that needs to be administered intrathecally, is approved for the treatment of 5q-spinal muscular atrophy (SMA). "
01/01/2023 - "Improvement of muscle strength in specific muscular regions in nusinersen-treated adult patients with 5q-spinal muscular atrophy."
01/01/2022 - "Ultrasound-guided transforaminal approach for nusinersen delivery in adult spinal muscle atrophy patients with challenging access."

01/01/2023 - "Treating neurodegenerative disease: Nusinersen and other therapeutic strategies for improved motor function."
01/01/2021 - "Nusinersen is a novel therapeutic approach with consistent results in all three studies and is proof of the novel concept for treating SMA and other neurodegenerative disorders in the future."
01/01/2021 - "Approval of nusinersen in 2016 demonstrated that effective treatments for neurodegenerative diseases can be identified and that treatments not only slow disease progression but also improve some symptoms. "
01/01/2021 - "An antisense oligonucleotide (ASO) drug, nusinersen, provides an upcoming and promising treatment option for SMA and represents a novel pharmacological approach with a mechanism of action relevant for other neurodegenerative disorders. "
01/01/2021 - "Spinal muscular atrophy is a progressive neurodegenerative disorder that can be treated with intrathecal antisense oligonucleotide therapy (nusinersen). "