Results 1 to 10 of about 3,114 (145)

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

Ann Clin Transl Neurol
ABSTRACT Objective Spinal Muscular Atrophy (SMA) is a rare genetic disorder marked by progressive muscle weakness and mobility loss. It has a profound physical, emotional and social impact on patients and caregivers, requiring comprehensive medical and supportive care.
Mehrabian A, Auguste P, Grove A, Brown A, Parr J, Patel M, Butt F, Donoghue J, Yousefi M, Parsons J.   +9 more
europepmc   +2 more sources

Nusinersen bei spinaler Muskelatrophie [PDF]

Nervenheilkunde, 2019
ZUSAMMENFASSUNGFür die 5q-assoziierte spinale Muskelatrophie (SMA) ist mit dem Antisense-Oligonukleotid Nusinersen seit Juni 2017 in Europa die erste kausale Therapie verfügbar. Die Zulassung beruht auf Phase-III-Studien bei Kindern und umfasst altersunabhängig alle genetisch gesicherten Subtypen der SMA. Erfahrungen in der Anwendung von Nusinersen bei
Olivia Schreiber-Katz, Susanne Petri, Friederich Götz, Omar Abu-Fares, Alma Osmanovic   +4 more
openaire   +1 more source

Nusinersen for SMA: expanded access programme [PDF]

Journal of Neurology, Neurosurgery & Psychiatry, 2018
BackgroundSpinal muscular atrophy (SMA) is a devastating motor neuron disorder causing progressive muscle weakness and respiratory insufficiency. We present the initial Australian experiences implementing the expanded access programme (EAP) to enable preapproval access to nusinersen, the first disease-modifying therapy, for SMA type 1.MethodsAn ...
Farrar, MA, Teoh, HL, Carey, KA, Cairns, A, Forbes, R, Herbert, K, Holland, S, Jones, KJ, Menezes, MP, Morrison, M, Munro, K, Villano, D, Webster, R, Woodcock, IR, Yiu, EM, Sampaio, H, Ryan, MM   +16 more
openaire   +3 more sources

Treatment of risdiplam after nusinersen continuously improves upper limb motor function in spinal muscular atrophy patients: a multicenter experience. [PDF]

Front Pediatr
Cheng X, Ma Y, Yu LQ, Fan YB, Zhu LH, Lu HB, Niu Q.   +6 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

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

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

Nusinersen for spinal muscular atrophy [PDF]

Therapeutic Advances in Neurological Disorders, 2018
Claudia D. Wurster, Albert C. Ludolph
openaire   +3 more sources

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

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