Results 111 to 120 of about 1,718 (175)

MyomiR Networks in Spinal Muscular Atrophy: Associations With Clinical Severity and Treatment Response. [PDF]

Mol Neurobiol
Barbo M, Koritnik B, Leonardis L, Dolžan V, Ravnik-Glavač M.   +4 more
europepmc   +1 more source

Preventing spinal muscular atrophy through the national premarital screening program in Türkiye: an economic comparison with treatment costs. [PDF]

Turk J Med Sci
Dur G, Tecirli G, Okumuş N.
europepmc   +1 more source

Elective preterm birth for earlier spinal muscular atrophy treatment. [PDF]

Mol Ther Adv
Dangouloff T, Chantraine F, Hennuy N, Rigo V, Tribolet S, Brande LV, Servais L.   +6 more
europepmc   +1 more source

Nusinersen: the antisense oligonucleotide at the forefront of spinal muscular atrophy treatment. [PDF]

RNA Biol
Singh NN, Ottesen EW, Singh RN.
europepmc   +1 more source

Safety and effectiveness of risdiplam in adults with spinal muscular atrophy: a systematic review. [PDF]

J Neurol
Alonge P, Urbano G, Gadaleta G.
europepmc   +1 more source

Clinical Experience of Timing Treatment in Newborns with Spinal Muscular Atrophy: A Call for Standardized Screening Practices in Italy. [PDF]

Int J Neonatal Screen
Bitetti I, Iannaccone R, Margiotta G, Varone A.   +3 more
europepmc   +1 more source

Pharmacokinetics of therapies approved for spinal muscular atrophy: A narrative review of current evidence. [PDF]

J Int Med Res
Sel EK, Gkrinia EMM, Roncato R, Vitezić D, Janković S, Belančić A, EACPT Early Career Clinical Pharmacologists Working Group.   +6 more
europepmc   +1 more source

Onasemnogene Abeparvovec in Early-Onset Spinal Muscular Atrophy: An Indian Experience. [PDF]

Ann Indian Acad Neurol
Desai N, Roy S, Coutinho F, Kasar U.
europepmc   +1 more source

Rare SMA Patients: A Comprehensive Look at Clinical Features, Genetic Profiles and Therapeutic Approaches. [PDF]

Int J Mol Sci
Mikhalchuk K, Artemieva S, Zabnenkova V, Akhkiamova M, Dadali E, Rudenskaya G, Sparber P, Rybakova O, Papina Y, Monakhova A, Shulyakova I, Saiko D, Zhiteneva S, Polyakov A, Shchagina O.   +14 more
europepmc   +1 more source

Economic evaluations of disease-modifying therapies for spinal muscular atrophy: a systematic literature review. [PDF]

Orphanet J Rare Dis
Yousefi M, Mehrabian A, Brown A, Butt F, Donoghue J, Parr J, Patel M, Grove A, Parsons J, Auguste P.   +9 more
europepmc   +1 more source