Skip to main page content
U.S. flag

An official website of the United States government

Dot gov

The .gov means it’s official.
Federal government websites often end in .gov or .mil. Before sharing sensitive information, make sure you’re on a federal government site.

Https

The site is secure.
The https:// ensures that you are connecting to the official website and that any information you provide is encrypted and transmitted securely.

Access keys NCBI Homepage MyNCBI Homepage Main Content Main Navigation

Search Page

Filters

My NCBI Filters

Results by year

Table representation of search results timeline featuring number of search results per year.

Year Number of Results
1951 1
1979 1
1982 1
1989 3
1990 1
1991 1
1992 2
1994 1
1996 1
1997 1
1998 1
2001 5
2002 8
2003 1
2004 9
2005 1
2006 3
2007 4
2008 4
2009 12
2010 10
2011 10
2012 11
2013 13
2014 9
2015 5
2016 9
2017 20
2018 31
2019 40
2020 42
2021 44
2022 47
2023 44
2024 23

Text availability

Article attribute

Article type

Publication date

Search Results

376 results

Results by year

Filters applied: . Clear all
Page 1
Efficacy and safety of onasemnogene abeparvovec for the treatment of patients with spinal muscular atrophy type 1: A systematic review with meta-analysis.
Fernandes BD, Krug BC, Rodrigues FD, Cirilo HNC, Borges SS, Schwartz IVD, Probst LF, Zimmermann I. Fernandes BD, et al. PLoS One. 2024 May 7;19(5):e0302860. doi: 10.1371/journal.pone.0302860. eCollection 2024. PLoS One. 2024. PMID: 38713659 Free PMC article.
BACKGROUND: Onasemnogene abeparvovec has been approved for the treatment of spinal muscular atrophy 5q type 1 in several countries, which calls for an independent assessment of the evidence regarding efficacy and safety. ...CONCLUSION: The results indi …
BACKGROUND: Onasemnogene abeparvovec has been approved for the treatment of spinal muscular atrophy 5q type 1 in …
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. Kokaliaris C, et al. Adv Ther. 2024 Jun;41(6):2414-2434. doi: 10.1007/s12325-024-02845-6. Epub 2024 May 5. Adv Ther. 2024. PMID: 38705943 Free PMC article.
INTRODUCTION: Spinal muscular atrophy (SMA) is a severe genetic neuromuscular disease characterized by a loss of motor neurons and progressive muscle weakness. ...Risdiplam and nusinersen are two approved treatments for patients with type 1 spinal
INTRODUCTION: Spinal muscular atrophy (SMA) is a severe genetic neuromuscular disease characterized by a loss of motor …
[Experience of the treatment of spinal muscular atrophy type 3 Kugelberg-Welander with Nusinersen].
Shchepankevich LA, Ushkalenko VK, Dolotov KA, Maksimova YV, Veretelnikov IA, Taneeva EV. Shchepankevich LA, et al. Zh Nevrol Psikhiatr Im S S Korsakova. 2024;124(4):153-158. doi: 10.17116/jnevro2024124041153. Zh Nevrol Psikhiatr Im S S Korsakova. 2024. PMID: 38676690 Russian.
Before the advent of pathogenetic therapy, the diagnosis of spinal muscular atrophy (SMA) meant the loss of all hopes for recovery and the patient's setting on the path of a steady decline in motor functions, a deterioration in the quality of life and, ultima …
Before the advent of pathogenetic therapy, the diagnosis of spinal muscular atrophy (SMA) meant the loss of all hopes f …
Nusinersen in adults with type 3 spinal muscular atrophy: long-term outcomes on motor and respiratory function.
Serrão C, Domingues S, de Campos CF, Moreira S, Conceição I, de Carvalho M, Oliveira Santos M. Serrão C, et al. Neurol Sci. 2024 Jun;45(6):2887-2891. doi: 10.1007/s10072-024-07515-7. Epub 2024 Apr 8. Neurol Sci. 2024. PMID: 38589769
INTRODUCTION: Nusinersen was approved for 5q spinal muscular atrophy (SMA), irrespective of age, SMA type or functional status. ...We aimed to characterize evolution on motor and respiratory function in our cohort of adults with type 3 SMA. METH …
INTRODUCTION: Nusinersen was approved for 5q spinal muscular atrophy (SMA), irrespective of age, SMA type or fun …
Neurophysiological Characteristics in Type II and Type III 5q Spinal Muscular Atrophy Patients: Impact of Nusinersen Treatment.
Li D, Sun N, Xiang L, Liu J, Wang X, Yang L, Huang S. Li D, et al. Drug Des Devel Ther. 2024 Mar 28;18:953-965. doi: 10.2147/DDDT.S449066. eCollection 2024. Drug Des Devel Ther. 2024. PMID: 38562520 Free PMC article.
OBJECTIVE: This study aimed to observe the neurophysiological characteristics of type II and type III 5q spinal muscular atrophy (SMA) patients and the changes in peripheral motor nerve electrophysiology after Nusinersen treatment, as wel …
OBJECTIVE: This study aimed to observe the neurophysiological characteristics of type II and type III 5q spinal
Global Risdiplam Compassionate Use Program for Patients with Type 1 or 2 Spinal Muscular Atrophy.
Kantaria R, Baker K, Beckley-Kartey S, Gorni K, Montrocher-Ober I, Vindevoghel L. Kantaria R, et al. Clin Ther. 2024 Apr;46(4):374-378. doi: 10.1016/j.clinthera.2024.02.006. Epub 2024 Mar 10. Clin Ther. 2024. PMID: 38461122 Free article.
PURPOSE: Spinal muscular atrophy (SMA) is a genetic neuromuscular disease causing progressive muscle weakness and reducing life expectancy. ...METHODS: Enrolled patients (aged 2 months) had type 1 or 2 SMA and no alternative treatment options (ie, they …
PURPOSE: Spinal muscular atrophy (SMA) is a genetic neuromuscular disease causing progressive muscle weakness and reduc …
Vaccination proposal for patients on onasemnogene abeparvovec therapy.
Dobner S, Kulcsár A, Liptai Z, Vojnisek Z, Constantin T, Szabó L. Dobner S, et al. Eur J Paediatr Neurol. 2024 Mar;49:95-99. doi: 10.1016/j.ejpn.2024.02.010. Epub 2024 Mar 1. Eur J Paediatr Neurol. 2024. PMID: 38457958 Free article.
The approval of disease-modifying treatment in spinal muscular atrophy made the condition less severe. The course of the disease changed, but some new concerns occurred with the different new therapies. ...
The approval of disease-modifying treatment in spinal muscular atrophy made the condition less severe. The course of th …
Disease Trajectories in the Revised Hammersmith Scale in a Cohort of Untreated Patients with Spinal Muscular Atrophy types 2 and 3.
Wolfe A, Stimpson G, Ramsey D, Coratti G, Dunaway Young S, Mayhew A, Pane M, Rohwer A, Muni Lofra R, Duong T, O'Reilly E, Milev E, Civitello M, Sansone VA, D'Amico A, Bertini E, Messina S, Bruno C, Albamonte E, Mazzone E, Main M, Montes J, Glanzman AM, Zolkipli-Cunningham Z, Pasternak A, Marini-Bettolo C, Day JW, Darras BT, De Vivo DC, Baranello G, Scoto M, Finkel RS, Mercuri E, Muntoni F; international SMA consortium (iSMAc). Wolfe A, et al. J Neuromuscul Dis. 2024;11(3):665-677. doi: 10.3233/JND-230211. J Neuromuscul Dis. 2024. PMID: 38427497 Free PMC article.
BACKGROUND: Spinal muscular atrophy (SMA) is a neuromuscular disorder characterised by progressive motor function decline. ...At baseline, this cohort included 50% females and 15% of patients had undergone spinal fusion surgery. Patient trajectories we …
BACKGROUND: Spinal muscular atrophy (SMA) is a neuromuscular disorder characterised by progressive motor function decli …
Risdiplam improves subjective swallowing quality in non-ambulatory adult patients with 5q-spinal muscular atrophy despite advanced motor impairment.
Brakemeier S, Lipka J, Schlag M, Kleinschnitz C, Hagenacker T. Brakemeier S, et al. J Neurol. 2024 May;271(5):2649-2657. doi: 10.1007/s00415-024-12203-9. Epub 2024 Feb 15. J Neurol. 2024. PMID: 38358553 Free PMC article.
BACKGROUND: 5q-associated spinal muscular atrophy (SMA) is characterized by the progressive loss of motor neurons with consecutive weakness and atrophy of the limb, respiratory, and bulbar muscles. ...METHODS: Bulbar function was evaluated prospectivel …
BACKGROUND: 5q-associated spinal muscular atrophy (SMA) is characterized by the progressive loss of motor neurons with …
Safety and Efficacy of Apitegromab in Patients With Spinal Muscular Atrophy Types 2 and 3: The Phase 2 TOPAZ Study.
Crawford TO, Darras BT, Day JW, Dunaway Young S, Duong T, Nelson LL, Barrett D, Song G, Bilic S, Cote S, Sadanowicz M, Iarrobino R, Xu TJ, O'Neil J, Rossello J, Place A, Kertesz N, Nomikos G, Chyung Y. Crawford TO, et al. Neurology. 2024 Mar 12;102(5):e209151. doi: 10.1212/WNL.0000000000209151. Epub 2024 Feb 8. Neurology. 2024. PMID: 38330285 Free PMC article. Clinical Trial.
BACKGROUND AND OBJECTIVES: Currently approved therapies for spinal muscular atrophy (SMA) reverse the degenerative course, leading to better functional outcome, but they do not address the impairment arising from preexisting neurodegeneration. ...CLASSIFICATI …
BACKGROUND AND OBJECTIVES: Currently approved therapies for spinal muscular atrophy (SMA) reverse the degenerative cour …
376 results