Results 121 to 130 of about 2,169 (197)

Real-world treatment patterns and unmet needs in spinal muscular atrophy: a caregiver-centric survey study from China. [PDF]

BMC Neurol
Fan W, Zheng WE, Li J, Wang R, Ni S, Xue P, Xu C.   +6 more
europepmc   +1 more source

SpliceHarmonization: an integrated method for identifying RNA splicing events in therapeutics for splicing modulation. [PDF]

Bioinformatics
Chen Y, Zhang H, Sun YH, Negi S, Cao S, Ouyang Z, Zhang B, Hurt J, Huh D.   +8 more
europepmc   +1 more source

Population Physiologically-Based Pharmacokinetic Modeling to Determine Ontogeny: A Quantitative Clinical Pharmacology Example in Pediatric Rare Disease. [PDF]

CPT Pharmacometrics Syst Pharmacol
Cleary Y, Prasad B, Ogungbenro K, Gertz M, Galetin A.   +4 more
europepmc   +1 more source

Correction: Matched-pair analysis of motor outcomes in adults with spinal muscular atrophy on nusinersen vs. risdiplam. [PDF]

J Neurol
Neuhoff S, Stolte B, Lipka J, Schlag M, Pul R, Schmitt LI, Leo M, Skuljec J, Deuschl C, Forsting M, Kleinschnitz C, Hagenacker T.   +11 more
europepmc   +1 more source

The effectiveness and value of therapies for spinal muscular atrophy. [PDF]

J Manag Care Spec Pharm
Tice JA, Luu L, Sanchez S, Lee W, Nikitin D, Phillips M, Carlson JJ, Chan HH, Rind DM, Agboola F.   +9 more
europepmc   +1 more source

Effectiveness of Risdiplam Treatment in Adult Patients With Spinal Muscular Atrophy Type IIb-III. [PDF]

Rev Neurol
García Estévez DA.
europepmc   +1 more source

Fibro-adipogenic progenitor cells from murine SMA muscles are intrinsically adipogenic. [PDF]

Proc Natl Acad Sci U S A
Luo YE, Abe-Teh Z, Alsaghir T, Heiden KD, Basso KB, Barton ER.   +5 more
europepmc   +1 more source

Drug Screening and Drug Repositioning as Promising Therapeutic Approaches for Spinal Muscular Atrophy Treatment [PDF]

, 2020
Boido, Marina Maria, Daniela Maria Rasà, Menduti, Giovanna, Stanga, Serena   +3 more
core   +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

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