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Clinical Evidence Supporting Early Treatment Of Patients With Spinal Muscular Atrophy: Current Perspectives.
Ther Clin Risk Manag 2019; 15:1153-1161TC

Abstract

Recent advances in the treatment of spinal muscular atrophy (SMA) have dramatically altered prognosis. Rather than a rapidly lethal disease, SMA type 1, the most severe form with the earliest onset of SMA, has become a disease in which long-term event-free survival with the acquisition of important motor milestones is likely. Prognosis for patients with SMA type 2 has shifted from slow and progressive deterioration to long-term stability. Nevertheless, there is a large heterogeneity in terms of clinical response to currently available treatments, ranging from absence of response to impressive improvement. The only factor identified that is predictive of treatment success is the age of the patient at the initiation of treatment, which is closely related to disease duration. The aim of this paper is to review available evidence that support early intervention using currently available treatment approaches.

Authors+Show Affiliations

Division of Child Neurology, Centre de Références des Maladies Neuromusculaires, Department of Pediatrics, University Hospital Liège & University of Liège, Liège, Belgium.Division of Child Neurology, Centre de Références des Maladies Neuromusculaires, Department of Pediatrics, University Hospital Liège & University of Liège, Liège, Belgium. MDUK Neuromuscular Center, Department of Paediatrics, University of Oxford, Oxford, UK.

Pub Type(s)

Journal Article

Language

eng

PubMed ID

31632042

Citation

Dangouloff, Tamara, and Laurent Servais. "Clinical Evidence Supporting Early Treatment of Patients With Spinal Muscular Atrophy: Current Perspectives." Therapeutics and Clinical Risk Management, vol. 15, 2019, pp. 1153-1161.
Dangouloff T, Servais L. Clinical Evidence Supporting Early Treatment Of Patients With Spinal Muscular Atrophy: Current Perspectives. Ther Clin Risk Manag. 2019;15:1153-1161.
Dangouloff, T., & Servais, L. (2019). Clinical Evidence Supporting Early Treatment Of Patients With Spinal Muscular Atrophy: Current Perspectives. Therapeutics and Clinical Risk Management, 15, pp. 1153-1161. doi:10.2147/TCRM.S172291.
Dangouloff T, Servais L. Clinical Evidence Supporting Early Treatment of Patients With Spinal Muscular Atrophy: Current Perspectives. Ther Clin Risk Manag. 2019;15:1153-1161. PubMed PMID: 31632042.
* Article titles in AMA citation format should be in sentence-case
TY - JOUR T1 - Clinical Evidence Supporting Early Treatment Of Patients With Spinal Muscular Atrophy: Current Perspectives. AU - Dangouloff,Tamara, AU - Servais,Laurent, Y1 - 2019/10/02/ PY - 2019/07/27/received PY - 2019/09/08/accepted PY - 2019/10/22/entrez PY - 2019/10/22/pubmed PY - 2019/10/22/medline KW - branaplam KW - newborn screening KW - nusinersen KW - risdiplam KW - spinal muscular atrophy KW - zolgensma SP - 1153 EP - 1161 JF - Therapeutics and clinical risk management JO - Ther Clin Risk Manag VL - 15 N2 - Recent advances in the treatment of spinal muscular atrophy (SMA) have dramatically altered prognosis. Rather than a rapidly lethal disease, SMA type 1, the most severe form with the earliest onset of SMA, has become a disease in which long-term event-free survival with the acquisition of important motor milestones is likely. Prognosis for patients with SMA type 2 has shifted from slow and progressive deterioration to long-term stability. Nevertheless, there is a large heterogeneity in terms of clinical response to currently available treatments, ranging from absence of response to impressive improvement. The only factor identified that is predictive of treatment success is the age of the patient at the initiation of treatment, which is closely related to disease duration. The aim of this paper is to review available evidence that support early intervention using currently available treatment approaches. SN - 1176-6336 UR - https://www.unboundmedicine.com/medline/citation/31632042/Clinical_Evidence_Supporting_Early_Treatment_Of_Patients_With_Spinal_Muscular_Atrophy:_Current_Perspectives L2 - https://dx.doi.org/10.2147/TCRM.S172291 DB - PRIME DP - Unbound Medicine ER -
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