Results 1 to 10 of about 4,206 (188)

Author Correction: 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

Serum creatinine to cystatin C ratio as a biomarker for monitoring motor-function in children with spinal muscular atrophy treated with nusinersen: a retrospective cohort study. [PDF]

BMC Neurol
Zhang YY, Wang J, Cui ZQ, Ma K.
europepmc   +1 more source

Economic evaluations of disease-modifying therapies for spinal muscular atrophy: a systematic literature review. [PDF]

Orphanet J Rare Dis
Yousefi M, Mehrabian A, Brown A, Butt F, Donoghue J, Parr J, Patel M, Grove A, Parsons J, Auguste P.   +9 more
europepmc   +1 more source

Risdiplam Impact in Treatment Naïve and Non-Naïve Pediatric and Adult Patients With Spinal Muscular Atrophy. [PDF]

Eur J Neurol
Yee POP, Ip HHN, Yu MKL, Cheng PK, Wong WHS, Ng PPH, Kwong NST, Lam P, Cheung MW, Tung A, Chan S, Ng S, Yeung WL, Wong S, Gao Y, Pang S, Chan SHS.   +16 more
europepmc   +1 more source

Effectiveness and Safety of Nusinersen and Risdiplam in Spinal Muscular Atrophy: A Systematic Review. [PDF]

Ann Clin Transl Neurol
Mehrabian A, Auguste P, Grove A, Brown A, Parr J, Patel M, Butt F, Donoghue J, Yousefi M, Parsons J.   +9 more
europepmc   +1 more source

Impact of Home-Based Respiratory Physiotherapy in Nusinersen-Treated Patients with Spinal Muscular Atrophy. [PDF]

Adv Ther
Be'er M, Shperling L, Rochman M, Amirav I, Cahal M, Lavi R, Sadot E, Lior Y, Lavie M.   +8 more
europepmc   +1 more source

Nusinersen Use in Spinal Muscular Atrophy [PDF]

Pediatrics, 2019
openaire   +2 more sources

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

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

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