Results 161 to 170 of about 19,843 (235)

Development and validation of a 4-color multiplexing spinal muscular atrophy (SMA) genotyping assay on a novel integrated digital PCR instrument. [PDF]

Sci Rep, 2020
Jiang L, Lin R, Gallagher S, Zayac A, Butchbach MER, Hung P.   +5 more
europepmc   +1 more source

Nusinersen versus sham control in later-onset spinal muscular atrophy [PDF]

, 2018
Connolly, A. M., et al,
core   +1 more source

Spinal Muscular Atrophy (SMA) Subtype Concordance in Siblings: Findings From the Cure SMA Cohort. [PDF]

J Neuromuscul Dis, 2020
Jones CC, Cook SF, Jarecki J, Belter L, Reyna SP, Staropoli J, Farwell W, Hobby K.   +7 more
europepmc   +1 more source

Spinal muscular atrophy (SMA).

Neurosciences (Riyadh, Saudi Arabia), 2011
Abdulaziz, Al-Saman, Osama, Mudhafar
openaire   +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. [PDF]

Hum Mol Genet
Hoolachan JM, McCallion E, Sutton ER, Çetin Ö, Pacheco-Torres P, Dimitriadi M, Sari S, Miller GJ, Okoh M, Walter LM, Claus P, Wood MJA, Tonge DP, Bowerman M.   +13 more
europepmc   +1 more source

The Need for SMN-Independent Treatments of Spinal Muscular Atrophy (SMA) to Complement SMN-Enhancing Drugs. [PDF]

Front Neurol, 2020
Hensel N, Kubinski S, Claus P.
europepmc   +1 more source

Onasemnogene Abeparvovec: Post-infusion Efficacy and Safety in Patients With Spinal Muscular Atrophy (SMA)-A Fondazione Policlinico Gemelli IRCCS Experience. [PDF]

Hosp Pharm
Favia M, Tarantino D, Cerbo LD, Sabia A, Campopiano R, Pani M.   +5 more
europepmc   +1 more source

Rights of Children Suffering from SMA (Spinal Muscular Atrophy) [PDF]

e-Pedagogium, 2010
openaire   +1 more source

Routine Cerebrospinal Fluid (CSF) Parameters in Patients With Spinal Muscular Atrophy (SMA) Treated With Nusinersen. [PDF]

Front Neurol, 2019
Wurster CD, Koch JC, Cordts I, Dreyhaupt J, Otto M, Uzelac Z, Witzel S, Winter B, Kocak T, Schocke M, Weydt P, Wollinsky K, Ludolph AC, Deschauer M, Lingor P, Tumani H, Hermann A, Günther R.   +17 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