Results 121 to 130 of about 641,821 (189)

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

Longitudinal multi-omics profiling of spinal muscular atrophy. [PDF]

Neurotherapeutics
Dabaj I, Nguyen THY, Lagrue E, Ducatez F, Allouche S, Ausseil J, Seferian A, Gomez-Garcia de la Banda M, Benezit A, Phelep A, Chouchane M, Vasseur S, Chapart M, Marret S, Quijano Roy S, Tebani A, Bekri S.   +16 more
europepmc   +1 more source

Unbiased bioinformatic and clinical analysis identifies glial fibrillary acidic protein as a potential biomarker in spinal muscular atrophy. [PDF]

Brain Commun
Soares de Oliveira KL, Weber AC, Becker MM, Magalhães JSA, de Albuquerque ALA, Chadanowicz JK, Giudicelli GC, Rohden F, Zimmer ER, Kowalski TW, Siebert M, Costa MCM, Saute JA.   +12 more
europepmc   +1 more source

Neonatal screening for spinal muscular atrophy: Report of a multicenter study in Brazil. [PDF]

Genet Mol Biol
Oliveira Netto AB, Hentschke-Lopes M, Pasetto FB, Anjos TARD, Andrade GS, Sousa MIA, Moraes IDS, Trapp FB, Brusius-Facchin AC, Saute JAM, Becker MM, Coutinho VLS, Vilella-Arias S, Ponci V, França Junior MC, Galera MF, Amorim T, Giugliani R.   +17 more
europepmc   +1 more source

Strengthening adaptive functioning and participation in the contemporary spinal muscular atrophy paradigm: clinical perspectives and future directions. [PDF]

World J Pediatr
Farah EE, Farrar MA, Kariyawasam DS, Paguinto SG.   +3 more
europepmc   +1 more source

Behavioral barriers in the management of spinal muscular atrophy: The role of procrastination, regret, and burnout. [PDF]

PLoS One
Maurino J, Carrera-García L, Castro-Fernández P, Málaga I, Nascimento A, Saposnik G.   +5 more
europepmc   +1 more source

Modeling the Budget Impact of Disease-Modifying Therapies in Infantile-Onset Spinal Muscular Atrophy. [PDF]

Clin Drug Investig
Abuloha S, Goodin A, Jiao T, Corti M, Svensson M.   +4 more
europepmc   +1 more source

Case Report: Presymptomatic risdiplam in preterm monozygotic twins with co-occurring spinal muscular atrophy and tuberous sclerosis complex. [PDF]

Front Genet
Zhang XF, Wang YJ, Li SX, Liu P, Hu WG.
europepmc   +1 more source