Articles: oligonucleotides.
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Patients with hereditary transthyretin (TTR) amyloidosis (hATTR) often experience disease progression after orthotopic liver transplant (POLT) due in part to wild type ATTR amyloid deposition. The management strategy is not defined. We propose that TTR gene silencing with an antisense oligonucleotide or a small interfering ribonucleic acid may be a treatment for these patients. ⋯ TTR gene silencing therapy in hATTR patients with POLT could be a treatment option. Vigilant monitoring of renal, liver and bone marrow functions is necessary because of frequent complications. Further studies are needed to determine efficacy.
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Diagn Interv Radiol · Nov 2020
Nusinersen injections in adults and children with spinal muscular atrophy: a single-center experience.
Nusinersen is a drug approved in December 2016 for treatment of spinal muscular atrophy (SMA). We want to share our initial experience with image-guided, non-image-guided, and port-delivered nusinersen injections in a large single-center SMA patient cohort, treating both pediatric and adult patients with focus on technical considerations and other patient concerns from a combined perspective of patient, neurologist, and radiologist. ⋯ With the introduction of nusinersen treatment, neurologists and radiologists play an important role in treatment of SMA patients and therefore should be familiar with different techniques and complications of drug administration. Using good technique, it is possible to have very low complication rates even in this complex patient population, and various image-guided procedures can be a safe alternative to surgical approach, even in the most difficult cases.
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J. Neurol. Neurosurg. Psychiatr. · Nov 2020
Nusinersen safety and effects on motor function in adult spinal muscular atrophy type 2 and 3.
To retrospectively investigate safety and efficacy of nusinersen in a large cohort of adult Italian patients with spinal muscular atrophy (SMA). ⋯ Our data provide further evidence of nusinersen safety and efficacy in adult SMA2 and SMA3, with the latter appearing to be cumulative over time. In patients with extremely advanced disease, effects on residual motor function are less clear.
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Data on combining molecular therapies that increase survival motor neuron protein for spinal muscular atrophy type 1 (SMA1) is lacking. ⋯ Combination molecular therapy is tolerated in SMA1 patients. Further studies are needed to determine whether there are circumstances in which combination therapy would be more efficacious than either monotherapy. Prolonged corticosteroid use and liver toxicity monitoring may be necessary with onasemnogene therapy.