Results 151 to 160 of about 19,843 (235)

Multi-omics profiling in spinal muscular atrophy (SMA): investigating lipid and metabolic alterations through longitudinal CSF analysis of Nusinersen-treated patients. [PDF]

J Neurol
Zandl-Lang M, Züllig T, Holzer M, Eichmann TO, Darnhofer B, Schwerin-Nagel A, Zobel J, Haidl H, Biebl A, Köfeler H, Plecko B.   +10 more
europepmc   +1 more source

Neurodegeneration Biomarkers in Adult Spinal Muscular Atrophy (SMA) Patients Treated with Nusinersen. [PDF]

Int J Mol Sci
Andrés-Benito P, Vázquez-Costa JF, Ñungo Garzón NC, Colomina MJ, Marco C, González L, Terrafeta C, Domínguez R, Ferrer I, Povedano M.   +9 more
europepmc   +1 more source

Design of a Non-Interventional Study to Validate a Set of Patient- and Caregiver-Oriented Measurements to Assess Health Outcomes in Spinal Muscular Atrophy (SMA-TOOL Study). [PDF]

Neurol Ther, 2021
Madruga-Garrido M, Vázquez-Costa JF, Medina-Cantillo J, Brañas M, Cattinari MG, de Lemus M, Díaz-Abós P, Sánchez-Menéndez V, Terrancle Á, Rebollo P, Maurino J.   +10 more
europepmc   +1 more source

Identification of the most relevant aspects of spinal muscular atrophy (SMA) with impact on the quality of life of SMA patients and their caregivers: the PROfuture project, a qualitative study. [PDF]

J Patient Rep Outcomes
de Lemus M, Cattinari MG, Pascual SI, Medina J, García M, Magallón A, Dumont M, Rebollo P.   +7 more
europepmc   +1 more source

A transcriptomics-based drug repositioning approach to identify drugs with similar activities for the treatment of muscle pathologies in spinal muscular atrophy (SMA) models

, 2023
Hoolachan JM, McCallion E, Sutton ER, Çetin Ö, Pacheco-Torres P, Dimitriadi M, Okoh M, Walter LM, Claus P, Wood MJ, Tonge DP, Bowerman M.   +11 more
europepmc   +1 more source

A Systematic Literature Review of the Natural History of Respiratory, Swallowing, Feeding, and Speech Functions in Spinal Muscular Atrophy (SMA). [PDF]

J Neuromuscul Dis
Martí Y, Aponte Ribero V, Batson S, Mitchell S, Gorni K, Gusset N, Oskoui M, Servais L, Deconinck N, McGrattan KE, Mercuri E, Sutherland CS.   +11 more
europepmc   +1 more source

Male Reproduction in Spinal Muscular Atrophy (SMA) and the Potential Impact of Oral Survival of Motor Neuron 2 (SMN2) Pre-mRNA Splicing Modifiers. [PDF]

Neurol Ther
Bar-Chama N, Elsheikh B, Hewamadduma C, Guittari CJ, Gorni K, Mueller L.   +5 more
europepmc   +1 more source

Assessment of Barriers to Referral and Appointment Wait Times for the Evaluation of Spinal Muscular Atrophy (SMA): Findings from a Web-Based Physician Survey. [PDF]

Neurol Ther
Curry MA, Cruz RE, Belter LT, Schroth MK, Jarecki J.   +4 more
europepmc   +1 more source

Peripheral drug delivery for spinal muscular atrophy (SMA) [PDF]

Science-Business eXchange, 2011
openaire   +1 more source

Design and Validation of a Clinical Outcome Measure for Adolescents and Adult Patients with Spinal Muscular Atrophy: SMA Life Study Protocol. [PDF]

Neurol Ther
Rebollo P, García-López S, Povedano M, Cattinari MG, Martínez-Moreno M, Terrancle Á, Cabello-Moruno R, Vázquez-Costa JF.   +7 more
europepmc   +1 more source