368 related articles for article (PubMed ID: 32727502)
1. Experiences of caregivers of children with spinal muscular atrophy participating in the expanded access program for nusinersen: a longitudinal qualitative study.
Kiefer P; Kirschner J; Pechmann A; Langer T
Orphanet J Rare Dis; 2020 Jul; 15(1):194. PubMed ID: 32727502
[TBL] [Abstract][Full Text] [Related]
2. Evaluation of Children with SMA Type 1 Under Treatment with Nusinersen within the Expanded Access Program in Germany.
Pechmann A; Langer T; Schorling D; Stein S; Vogt S; Schara U; Kölbel H; Schwartz O; Hahn A; Giese K; Johannsen J; Denecke J; Weiß C; Theophil M; Kirschner J
J Neuromuscul Dis; 2018; 5(2):135-143. PubMed ID: 29689734
[TBL] [Abstract][Full Text] [Related]
3. Nusinersen treatment of Spinal Muscular Atrophy Type 1 - results of expanded access programme in Poland.
Modrzejewska S; Kotulska K; Kopyta I; Grędowska E; Emich-Widera E; Tomaszek K; Paprocka J; Chmielewski D; Pilch J; Pietruszewski J; Lemska A; Zawadzka M; Mazurkiewicz-Bełdzińska M
Neurol Neurochir Pol; 2021; 55(3):289-294. PubMed ID: 33565602
[TBL] [Abstract][Full Text] [Related]
4. Implementing a Global Expanded Access Program (EAP) for Infantile-Onset Spinal Muscular Atrophy (Type I): Understanding the Imperative, Impact and Challenges.
Yong J; Moffett M; Lucas S
J Neuromuscul Dis; 2019; 6(2):227-231. PubMed ID: 31127730
[TBL] [Abstract][Full Text] [Related]
5. Long-Term Comparative Efficacy and Safety of Risdiplam and Nusinersen in Children with Type 1 Spinal Muscular Atrophy.
Kokaliaris C; Evans R; Hawkins N; Mahajan A; Scott DA; Sutherland CS; Nam J; Sajeev G
Adv Ther; 2024 Jun; 41(6):2414-2434. PubMed ID: 38705943
[TBL] [Abstract][Full Text] [Related]
6. Perspectives on Spinraza (Nusinersen) Treatment Study: Views of Individuals and Parents of Children Diagnosed with Spinal Muscular Atrophy.
Pacione M; Siskind CE; Day JW; Tabor HK
J Neuromuscul Dis; 2019; 6(1):119-131. PubMed ID: 30594933
[TBL] [Abstract][Full Text] [Related]
7. A comprehensive institutional overview of intrathecal nusinersen injections for spinal muscular atrophy.
Mousa MA; Aria DJ; Schaefer CM; Kaye RD; Abruzzo TA; Bernes SM; Willard SD; Riemann MC; Towbin RB
Pediatr Radiol; 2018 Nov; 48(12):1797-1805. PubMed ID: 30022258
[TBL] [Abstract][Full Text] [Related]
8. Understanding caregiver experiences with disease-modifying therapies for spinal muscular atrophy: a qualitative study.
Xiao L; Kang S; Djordjevic D; Gonorazky H; Chiang J; Ambreen M; Nigro E; Law E; Weinstock L; McCradden M; Amin R
Arch Dis Child; 2023 Nov; 108(11):929-934. PubMed ID: 37419673
[TBL] [Abstract][Full Text] [Related]
9. Nusinersen for SMA: expanded access programme.
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
J Neurol Neurosurg Psychiatry; 2018 Sep; 89(9):937-942. PubMed ID: 29549190
[TBL] [Abstract][Full Text] [Related]
10. Nusinersen for Spinal Muscular Atrophy in the United States: Findings From a Retrospective Claims Database Analysis.
Gauthier-Loiselle M; Cloutier M; Toro W; Patel A; Shi S; Davidson M; Bischof M; LaMarca N; Dabbous O
Adv Ther; 2021 Dec; 38(12):5809-5828. PubMed ID: 34713391
[TBL] [Abstract][Full Text] [Related]
11. Early experiences of nusinersen for the treatment of spinal muscular atrophy: Results from a large survey of patients and caregivers.
Chen E; Dixon S; Naik R; Noone JM; Buchenberger JD; Whitmire SM; Mills R; Arnold W
Muscle Nerve; 2021 Mar; 63(3):311-319. PubMed ID: 33184859
[TBL] [Abstract][Full Text] [Related]
12. Children and young adults with spinal muscular atrophy treated with nusinersen.
Osredkar D; Jílková M; Butenko T; Loboda T; Golli T; Fuchsová P; Rohlenová M; Haberlova J
Eur J Paediatr Neurol; 2021 Jan; 30():1-8. PubMed ID: 33307321
[TBL] [Abstract][Full Text] [Related]
13. Nusinersen induces detectable changes in compound motor action potential response in spinal muscular atrophy type 1 patients with severe impairment of motor function.
Ueda Y; Egawa K; Kawamura K; Ochi N; Goto T; Kimura S; Narugami M; Nakakubo S; Nakajima M; Manabe A; Shiraishi H
Brain Dev; 2024 Mar; 46(3):149-153. PubMed ID: 38103972
[TBL] [Abstract][Full Text] [Related]
14. Efficacy of nusinersen in type 1, 2 and 3 spinal muscular atrophy: Real world data from Hungarian patients.
Szabó L; Gergely A; Jakus R; Fogarasi A; Grosz Z; Molnár MJ; Andor I; Schulcz O; Goschler Á; Medveczky E; Czövek D; Herczegfalvi Á
Eur J Paediatr Neurol; 2020 Jul; 27():37-42. PubMed ID: 32456992
[TBL] [Abstract][Full Text] [Related]
15. Adherence and Persistence to Nusinersen for Spinal Muscular Atrophy: A US Claims-Based Analysis.
Fox D; To TM; Seetasith A; Patel AM; Iannaccone ST
Adv Ther; 2023 Mar; 40(3):903-919. PubMed ID: 36534265
[TBL] [Abstract][Full Text] [Related]
16. Gene replacement therapy with onasemnogene abeparvovec in children with spinal muscular atrophy aged 24 months or younger and bodyweight up to 15 kg: an observational cohort study.
Weiß C; Ziegler A; Becker LL; Johannsen J; Brennenstuhl H; Schreiber G; Flotats-Bastardas M; Stoltenburg C; Hartmann H; Illsinger S; Denecke J; Pechmann A; Müller-Felber W; Vill K; Blaschek A; Smitka M; van der Stam L; Weiss K; Winter B; Goldhahn K; Plecko B; Horber V; Bernert G; Husain RA; Rauscher C; Trollmann R; Garbade SF; Hahn A; von der Hagen M; Kaindl AM
Lancet Child Adolesc Health; 2022 Jan; 6(1):17-27. PubMed ID: 34756190
[TBL] [Abstract][Full Text] [Related]
17. Nusinersen for the treatment of spinal muscular atrophy.
Chiriboga CA
Expert Rev Neurother; 2017 Oct; 17(10):955-962. PubMed ID: 28884620
[TBL] [Abstract][Full Text] [Related]
18. A mixed method study on the impact of living with spinal muscular atrophy in Malaysia from patients' and caregivers' perspectives.
Ch'ng GS; Koh K; Ahmad-Annuar A; Taib F; Koh CL; Lim ESC
Orphanet J Rare Dis; 2022 May; 17(1):200. PubMed ID: 35578241
[TBL] [Abstract][Full Text] [Related]
19. The effects of nusinersen treatment on respiratory status of children with spinal muscular atrophy.
Hepkaya E; Kılınç Sakallı AA; Ülkersoy İ; Başkan AK; Arslan H; Meral Ö; Dilek TD; Güler S; Saltık S; Çokuğraş H
Pediatr Int; 2022 Jan; 64(1):e15310. PubMed ID: 36310036
[TBL] [Abstract][Full Text] [Related]
20. An Integrated Safety Analysis of Infants and Children with Symptomatic Spinal Muscular Atrophy (SMA) Treated with Nusinersen in Seven Clinical Trials.
Darras BT; Farrar MA; Mercuri E; Finkel RS; Foster R; Hughes SG; Bhan I; Farwell W; Gheuens S
CNS Drugs; 2019 Sep; 33(9):919-932. PubMed ID: 31420846
[TBL] [Abstract][Full Text] [Related]
[Next] [New Search]