Results 121 to 130 of about 1,977 (181)

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

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

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

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

Cognitive and neurodevelopmental outcomes in spinal muscular atrophy: a scoping review. [PDF]

Front Cell Neurosci
Gnazzo M, Pisanò G, Baldini V, Giordani S, Caiazza L, Piccolo B, Turco E, Esposito S, Pera MC.   +8 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

Addressing the Global Disparities in Access to Treatment in Spinal Muscular Atrophy. [PDF]

Muscle Nerve
Koutsioumpa C, Darras BT, Griggs RC, Roy B.   +3 more
europepmc   +1 more source

Onasemnogene Abeparvovec in Patients With SMA: Interim Results of the RESTORE Registry in Japan. [PDF]

Ann Clin Transl Neurol
Saito K, Benguerba K, Tsuchida K, Yazawa K, Tsumiyama I, Kayama H, Reyna SP, Khan F, Finkel RS.   +8 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

The evolving therapeutic landscape of spinal muscular atrophy - A scoping review of investigational agents, emerging delivery technologies and strategic innovations. [PDF]

Br J Clin Pharmacol
Belančić A, Eustaquio P, Gkrinia EMM, Stević I, Javor E, Lam YW, Janković S, Vitezić D.   +7 more
europepmc   +1 more source