Potential disease-modifying therapies for Huntington's disease: lessons learned and future opportunities
- Biology, MedicineThe Lancet Neurology
Huntington's disease clinical trials corner: April 2022.
- Medicine, PhysicsJournal of Huntington's disease
In this edition of the Huntington's Disease Clinical Trials Corner we expand on GENERATION HD1, PRECISION-HD1 and PRECISION-HD2, SELECT-HD, and VIBRANT-HD trials, and list all currently registered…
Nusinersen treatment significantly improves hand grip strength, hand motor function and MRC sum scores in adult patients with spinal muscular atrophy types 3 and 4
- Medicine, BiologyJournal of Neurology
It is concluded that hand grip strength and hand motor function, as well as MRC sum scores improved significantly in nusinersen-treated adult patients with SMA types 3 and 4.
Routine Cerebrospinal Fluid (CSF) Parameters in Patients With Spinal Muscular Atrophy (SMA) Treated With Nusinersen
- Medicine, BiologyFront. Neurol.
The data suggest that a regular examination of routine CSF parameters in patients in which intrathecal ASOs are administered is important to obtain information on possible side effects and to gain further insights into intr Athecal processes.
Reader response: Evidence in focus: Nusinersen use in spinal muscular atrophy: Report of the Guideline Development, Dissemination, and Implementation Subcommittee of the American Academy of Neurology
A patient with SMA type 1 showing communicating hydrocephalus related to nusinersen treatment is reported, and it is reported that this patient is the first in the United States to be shown to have this condition after treatment with this drug.
Genetic neuromuscular disorders: living the era of a therapeutic revolution. Part 2: diseases of motor neuron and skeletal muscle
- Biology, MedicineNeurological Sciences
The present review is for diseases of motor neuron and skeletal muscle, some of which reached recently the most innovative therapeutic approaches, including gene therapy in X-linked myotubular myopathy and Pompe disease, and antisense oligonucleotides in myotonic dystrophy type 1.
Evidence in focus: Nusinersen use in spinal muscular atrophy
The level of evidence for use of nusinersen to treat spinal muscular atrophy (SMA) is identified and clinical considerations regarding use are reviewed, with evidence of efficacy currently highest for treatment of infantile- and childhood-onset SMA in the early and middle symptomatic phases.