These tools will no longer be maintained as of December 31, 2024. Archived website can be found here. PubMed4Hh GitHub repository can be found here. Contact NLM Customer Service if you have questions.
281 related articles for article (PubMed ID: 33104036)
41. RESTORE: A Prospective Multinational Registry of Patients with Genetically Confirmed Spinal Muscular Atrophy - Rationale and Study Design. Finkel RS; Day JW; De Vivo DC; Kirschner J; Mercuri E; Muntoni F; Shieh PB; Tizzano E; Desguerre I; Quijano-Roy S; Saito K; Droege M; Dabbous O; Khan F; Renault L; Anderson FA; Servais L J Neuromuscul Dis; 2020; 7(2):145-152. PubMed ID: 32039859 [TBL] [Abstract][Full Text] [Related]
42. Economic burden of spinal muscular atrophy in the United States: a contemporary assessment. Droege M; Sproule D; Arjunji R; Gauthier-Loiselle M; Cloutier M; Dabbous O J Med Econ; 2020 Jan; 23(1):70-79. PubMed ID: 31322019 [No Abstract] [Full Text] [Related]
43. Molecular analysis of candidate genes on chromosome 5q13 in autosomal recessive spinal muscular atrophy: evidence of homozygous deletions of the SMN gene in unaffected individuals. Hahnen E; Forkert R; Marke C; Rudnik-Schöneborn S; Schönling J; Zerres K; Wirth B Hum Mol Genet; 1995 Oct; 4(10):1927-33. PubMed ID: 8595417 [TBL] [Abstract][Full Text] [Related]
44. A natural history study of late onset spinal muscular atrophy types 3b and 4. Piepers S; van den Berg LH; Brugman F; Scheffer H; Ruiterkamp-Versteeg M; van Engelen BG; Faber CG; de Visser M; van der Pol WL; Wokke JH J Neurol; 2008 Sep; 255(9):1400-4. PubMed ID: 18575920 [TBL] [Abstract][Full Text] [Related]
45. Correlation between SMN2 copy number and clinical phenotype of spinal muscular atrophy: three SMN2 copies fail to rescue some patients from the disease severity. Harada Y; Sutomo R; Sadewa AH; Akutsu T; Takeshima Y; Wada H; Matsuo M; Nishio H J Neurol; 2002 Sep; 249(9):1211-9. PubMed ID: 12242541 [TBL] [Abstract][Full Text] [Related]
46. Prevalence of Spinal Muscular Atrophy in the Era of Disease-Modifying Therapies: An Italian Nationwide Survey. Coratti G; Ricci M; Capasso A; D'amico A; Sansone V; Bruno C; Messina S; Ricci F; Mongini T; Coccia M; Siciliano G; Pegoraro E; Turri M; Filosto M; Comi G; Masson R; Maggi L; Bruno I; D'Angelo MG; Trabacca A; Vacchiano V; Donati M; Simone I; Ruggiero L; Varone A; Verriello L; Berardinelli A; Agosto C; Pini A; Maioli MA; Passamano L; Brighina F; Carboni N; Garibaldi M; Zuccarino R; Gagliardi D; Siliquini S; Previtali S; Taruscio D; Boccia S; Pera MC; Pane M; Mercuri E; Neurology; 2023 Mar; 100(11):522-528. PubMed ID: 36460469 [TBL] [Abstract][Full Text] [Related]
47. Systematic literature review of the economic burden of spinal muscular atrophy and economic evaluations of treatments. Dangouloff T; Botty C; Beaudart C; Servais L; Hiligsmann M Orphanet J Rare Dis; 2021 Jan; 16(1):47. PubMed ID: 33485382 [TBL] [Abstract][Full Text] [Related]
48. [Infantile spinal atrophy: our experience in the last 25 years]. Madrid Rodríguez A; Martínez Martínez PL; Ramos Fernández JM; Urda Cardona A; Martínez Antón J An Pediatr (Barc); 2015 Mar; 82(3):159-65. PubMed ID: 25103837 [TBL] [Abstract][Full Text] [Related]
49. "Getting ready for the adult world": how adults with spinal muscular atrophy perceive and experience healthcare, transition and well-being. Wan HWY; Carey KA; D'Silva A; Kasparian NA; Farrar MA Orphanet J Rare Dis; 2019 Apr; 14(1):74. PubMed ID: 30940178 [TBL] [Abstract][Full Text] [Related]
50. Natural history of denervation in SMA: relation to age, SMN2 copy number, and function. Swoboda KJ; Prior TW; Scott CB; McNaught TP; Wride MC; Reyna SP; Bromberg MB Ann Neurol; 2005 May; 57(5):704-12. PubMed ID: 15852397 [TBL] [Abstract][Full Text] [Related]
51. Understanding the experiences and needs of individuals with Spinal Muscular Atrophy and their parents: a qualitative study. Qian Y; McGraw S; Henne J; Jarecki J; Hobby K; Yeh WS BMC Neurol; 2015 Oct; 15():217. PubMed ID: 26499462 [TBL] [Abstract][Full Text] [Related]
52. Prenusinersen economic and health-related quality of life burden of spinal muscular atrophy. Chambers GM; Settumba SN; Carey KA; Cairns A; Menezes MP; Ryan M; Farrar MA Neurology; 2020 Jul; 95(1):e1-e10. PubMed ID: 32513788 [TBL] [Abstract][Full Text] [Related]
53. The International Classification of Functioning, Disability and Health-Children and Youth as a framework for the management of spinal muscular atrophy in the era of gene therapy: a proof-of-concept study. Trabacca A; Lucarelli E; Pacifico R; Vespino T; Di Liddo A; Losito L Eur J Phys Rehabil Med; 2020 Apr; 56(2):243-251. PubMed ID: 31939268 [TBL] [Abstract][Full Text] [Related]
54. 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]
55. 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]
56. Quality of life of children with spinal muscular atrophy and their caregivers from the perspective of caregivers: a Chinese cross-sectional study. Yao M; Ma Y; Qian R; Xia Y; Yuan C; Bai G; Mao S Orphanet J Rare Dis; 2021 Jan; 16(1):7. PubMed ID: 33407670 [TBL] [Abstract][Full Text] [Related]
57. Rasch analysis of clinical outcome measures in spinal muscular atrophy. Cano SJ; Mayhew A; Glanzman AM; Krosschell KJ; Swoboda KJ; Main M; Steffensen BF; Bérard C; Girardot F; Payan CA; Mercuri E; Mazzone E; Elsheikh B; Florence J; Hynan LS; Iannaccone ST; Nelson LL; Pandya S; Rose M; Scott C; Sadjadi R; Yore MA; Joyce C; Kissel JT; Muscle Nerve; 2014 Mar; 49(3):422-30. PubMed ID: 23836324 [TBL] [Abstract][Full Text] [Related]
58. Intragenic mutations in SMN1 may contribute more significantly to clinical severity than SMN2 copy numbers in some spinal muscular atrophy (SMA) patients. Yamamoto T; Sato H; Lai PS; Nurputra DK; Harahap NI; Morikawa S; Nishimura N; Kurashige T; Ohshita T; Nakajima H; Yamada H; Nishida Y; Toda S; Takanashi J; Takeuchi A; Tohyama Y; Kubo Y; Saito K; Takeshima Y; Matsuo M; Nishio H Brain Dev; 2014 Nov; 36(10):914-20. PubMed ID: 24359787 [TBL] [Abstract][Full Text] [Related]
59. 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]
60. Telemedicine Use, Comfort, and Perceived Effectiveness in the Spinal Muscular Atrophy Community. Peterson IS; Belter LT; Curry MA; Jarecki J Telemed J E Health; 2024 Feb; 30(2):536-544. PubMed ID: 37566530 [No Abstract] [Full Text] [Related] [Previous] [Next] [New Search]