Results 111 to 120 of about 3,114 (145)

Patient reported outcome measures in spinal muscular atrophy and duchenne muscular dystrophy: review of instruments and their inclusion in clinical and regulatory processes. [PDF]

Neurol Sci
Malandrini F, Spataro C, Meregaglia M, Sansone V, D'amico A, Scopinaro A, Ciani O.   +6 more
europepmc   +1 more source

Evaluating Therapeutic Outcomes in Spinal Muscular Atrophy: An Indian Experience. [PDF]

Ann Indian Acad Neurol
Mohanlal S, Mubeena PM, Hussain H, Anand M, Kumar S, Ashraf VV, Rabia TA, Janardhanan S, Kumar S.   +8 more
europepmc   +1 more source

Cerebrospinal fluid metabolomics reveals predictive biomarkers of nusinersen therapy efficacy in type II and type III spinal muscular atrophy patients. [PDF]

Neurol Sci
Li D, Sun N, Wu H, Wang X, Shi Y, Yang L, Huang S, Zhang K, Yang C.   +8 more
europepmc   +1 more source

Machine learning predicts treatment response to nusinersen in non-sitter Spinal Muscular Atrophy (SMA).

Stimpson G, O'Reilly E, Coratti G, Ridout D, Chakraborti T, Mitra R, de Sanctis R, Pane M, Scoto M, Mercuri E, Muntoni F, Baranello G, International SMA Consortium.   +12 more
europepmc   +1 more source

Cost-effectiveness of treatments for presymptomatic newborn patients with spinal muscular atrophy and two or three copies of the survival motor neuron 2 gene in Italy. [PDF]

Eur J Health Econ
Valentini I, Ghetti G, Pane M, Rumi F, Di Brino E, Basile M, Pistillo GM, Bischof M.   +7 more
europepmc   +1 more source

Difficulties of Eating and Masticating Solid Food in Children with Spinal Muscular Atrophy-Preliminary Study. [PDF]

Nutrients
Winnicka E, Łabuz A, Kułaga Z, Grochowski T, Socha P.   +4 more
europepmc   +1 more source

Nusinersen corrects L-arginine deficiency in the cerebrospinal fluid of patients with severe spinal muscular atrophy. [PDF]

Neurobiol Dis
Hassan A, di Vito R, Caretto A, Nuzzo T, D'Amico A, Panicucci C, Bruno C, Bertini E, Vercelli A, Boido M, Errico F, Pellizzoni L, Usiello A.   +12 more
europepmc   +1 more source

Systematic Review and Meta-analysis of Long-Term Nusinersen Effectiveness in Adolescents and Adults with Spinal Muscular Atrophy. [PDF]

Adv Ther
Hagenacker T, Paradis AD, Lawson-Michod KA, Youn B.   +3 more
europepmc   +1 more source

Biomarkers in spinal muscular atrophy. [PDF]

Front Neurol
Yan L, Zhang J, Zheng J, Hao H.
europepmc   +1 more source

Extracellular Matrix Remodeling in Motor Neuron Diseases. [PDF]

Int J Mol Sci
Apolloni S, Tortoriello S, Milani M, Rossi S.   +3 more
europepmc   +1 more source