Results 121 to 130 of about 4,206 (188)

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

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

Treatment evolution in spinal muscular atrophy: insights from the SMArtCARE registry. [PDF]

Brain
Voigt-Müller C, Pfaffenlehner M, Bernert G, Cetin H, Hagenacker T, Kölbel H, Lochmüller H, Pfeuffer C, Vill K, Walter MC, Kirschner J, Pechmann A, SMArtCARE study group .   +12 more
europepmc   +1 more source

Electrophysiological Changes in Pediatric Spinal Muscular Atrophy: Results From an Observational Study. [PDF]

Muscle Nerve
Sun R, Wang J, Han J, Jiang J, Wu J, Chen X, Deng X, Cai C, Sun D.   +8 more
europepmc   +1 more source

High-dose nusinersen for spinal muscular atrophy: a phase 3 randomized trial. [PDF]

Nat Med
Finkel RS, Crawford TO, Mercuri E, Sumner CJ, Garcia Romero MDM, Day JW, Montes J, Sun P, Tichler B, Paradis AD, Boesch E, Inra J, Littauer R, Sohn J, Monine M, Gambino G, Foster R, Farewell R, Fradette S.   +18 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

Optimizing Oligonucleotide Therapeutics: A Model-Informed Drug Development Perspective. [PDF]

Clin Transl Sci
Yuan Y, Sharma V, Bhattaram VA, Pan X, Earp J, Wang Y, Liu J, Zhu H.   +7 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

[Spinal muscular atrophy: Clinical and genetic aspects, and therapeutic alternatives]. [PDF]

Rev Med Inst Mex Seguro Soc
González-Morales IJ, Olivera-Bernal GC, León-González C DA, Rosas-Vargas H.   +3 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