Results 141 to 150 of about 7,657 (243)

RNA biomarkers in spinal muscular atrophy: enhancing pathogenesis understanding and guiding precision medicine. [PDF]

Cell Mol Life Sci
Alberti C, Berardinelli A, Comi GP, Ottoboni L, Corti S.   +4 more
europepmc   +1 more source

Swallowing and feeding after disease-modifying treatment for spinal muscular atrophy: a systematic review of assessment modalities and outcomes. [PDF]

Orphanet J Rare Dis
Martí Y, Gorni K, Kumari S, Mahajan A, Baranello G, Waele L, McGrattan KE.   +6 more
europepmc   +1 more source

Assessment of safety and efficacy of risdiplam treatment in adults with spinal muscular atrophy. [PDF]

Front Neurol
Jaworek A, Jira K, Allen M, Zhao S, Kelly K, Moravec T, Tellez M, Heintzman S, Reynolds J, Sterling G, Kolb SJ, Arnold WD, Elsheikh B.   +12 more
europepmc   +1 more source

A real-world, multicentre, epidemiological study in Czech and Slovak adults with spinal muscular atrophy treated with risdiplam. [PDF]

Sci Rep
Parmova O, Prasil K, Mokra L, Svoboda M, Ridzon P, Junkerova J, Bakosova L, Okalova K, Jurikova L, Kolnikova M, Haberlova J.   +10 more
europepmc   +1 more source

Quantitative Whole-Body Muscle MRI in Adults With Spinal Muscular Atrophy-A Sensitive Tool for Long-Time Evaluation of Disease Progression. [PDF]

Eur J Neurol
Mensch A, Troppa B, Schneider I, Stapf CD, Koelsch AK, Kendzierski T, Strube D, Plutz S, Obenauf M, Delank KS, Kraya T, Otto M, Stoevesandt D, Naegel S.   +13 more
europepmc   +1 more source

Spontaan spinaal epiduraal hematoom bij acute rugpijn [PDF]

, 2017
Anisau, Aliaksandr, Vangeneugden, Johan, Vanhoenacker, Filip   +2 more
core   +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

Antisense oligonucleotides extend survival and reverse decrement in muscle response in ALS models [PDF]

, 2018
et al,, Hoye, Mariah L, Miller, Timothy M, Schoch, Kathleen M, Setnicka, Amy, Shabsovich, Mark, Wegener, Amy J   +6 more
core   +1 more source

Reply to the Comment by Zhang et al. [PDF]

Eur J Neurol
Bjelica B.
europepmc   +1 more source

Nusinersen Use in Spinal Muscular Atrophy [PDF]

Pediatrics, 2019
openaire   +2 more sources