Results 251 to 260 of about 7,543 (282)

Nusinersen treatment response markers

Nature Reviews Neurology, 2022
openaire   +2 more sources

Ergebnisse zu Nusinersen [PDF]

InFo Neurologie & Psychiatrie, 2018
openaire   +1 more source

Predictive Clinical-Biological Markers Over the First 3 Years of Nusinersen Treatment in SMA Type 1 Patients

Balneo and PRM Research Journal
Werdnig-Hoffmann disease, or type 1 Spinal Muscular Atrophy (SMA), is caused by insufficient SMN protein synthesis due to a genetic defect. Symptoms appear within the first 6 months of life, and without ventilatory support, life expectancy averages 2 ...
Mihaela Bădina, A. Mirea, Corina Sporea, G. Bejan   +3 more
semanticscholar   +1 more source

Plecanatide, Nusinersen, and Obeticholic acid

Journal of the American Pharmacists Association, 2017
Daniel A. Hussar, Deborah K. Douglas
openaire   +3 more sources

Evolution of Functional and Paraclinical Markers as Predictive Factors in Pediatric Late-Onset SMA Under Nusinersen Treatment: The Role of CSF pNF-H

Balneo and PRM Research Journal
Spinal Muscular Atrophy (SMA) is a rare neurodegenerative disease caused by insufficient synthesis of SMN protein, characterized by progressive muscle weakness, atrophy, and complications affecting the respiratory and digestive systems.
Mihaela Bădina, G. Bejan, Corina Sporea, A. Mirea   +3 more
semanticscholar   +1 more source

Nusinersen, an antisense oligonucleotide drug for spinal muscular atrophy

Nature Neuroscience, 2017
Nusinersen (Spinraza) is a recently approved drug for treating spinal muscular atrophy. Approval of nusinersen may signal new opportunities for using antisense oligonucleotides as treatments for devastating neurological diseases.
openaire   +2 more sources

A Case Series Examining Clinic, Laboratory, and Physical Function After Administration of Nusinersen in Adults With Spinal Muscular Atrophy, a Single-Center Study

Journal of Clinical Neuromuscular Disease
Supplemental Digital Content is Available in the Text. Abstract Spinal muscular atrophy is an incurable inherited disease caused by lower motor neuron death from mutations of the survival motor neuron genes.
Tulio Bertorini, Janna Knickerbocker, Hagar Anwar, William H. Mays, Kimberly Carter, E. Metter, Laura Talbot   +6 more
semanticscholar   +1 more source

A CSF marker of nusinersen treatment response

Nature Reviews Neurology, 2022
openaire   +2 more sources

Nusinersen treatment of Spinal Muscular Atrophy Type 1 — results of expanded access programme in Poland

Neurologia I Neurochirurgia Polska, 2021
Sandra Modrzejewska, Anna Lemska, Ilona Kopyta   +2 more
exaly  

Intrathecal nusinersen administration: Is anesthesia really needed?

Pediatric Anesthesia, 2021
Anna Camporesi, Anna Mandelli, Elisa Melloni   +2 more
openaire   +3 more sources