Early experiences of nusinersen for the treatment of spinal muscular atrophy: Results from a large survey of patients and caregivers.

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Abstrakt

Tytuł:: Early experiences of nusinersen for the treatment of spinal muscular atrophy: Results from a large survey of patients and caregivers.
Autorzy:: Chen E; Genentech Inc, San Francisco, California, USA.
Dixon S; Department of Neurology, University of Colorado School of Medicine, Aurora, Colorado, USA.
Naik R; Noesis Healthcare Technologies, Inc, San Francisco, California, USA.
Noone JM; Ipsos Healthcare, New York, New York, USA.
Buchenberger JD; Ipsos Healthcare, New York, New York, USA.
Whitmire SM; Ipsos Healthcare, New York, New York, USA.
Mills R; Ipsos Healthcare, New York, New York, USA.
Arnold W; Department of Neurology, The Ohio State University, Columbus, Ohio, USA.
Źródło:: Muscle & nerve [Muscle Nerve] 2021 Mar; Vol. 63 (3), pp. 311-319. Date of Electronic Publication: 2020 Dec 19.
Typ publikacji:: Journal Article; Research Support, Non-U.S. Gov't
Język:: English
Imprint Name(s):: Publication: <2005-> : Hoboken, NJ : John Wiley & Sons
Original Publication: New York, NY : John Wiley & Sons
MeSH Terms:: Caregivers*
Patient Satisfaction*
Muscular Atrophy, Spinal/*drug therapy
Oligonucleotides/*therapeutic use
Activities of Daily Living ; Adolescent ; Adult ; Child ; Child, Preschool ; Female ; Health Expenditures ; Humans ; Infant ; Injections, Spinal ; Insurance Coverage ; Insurance, Health ; Male ; Middle Aged ; Muscular Atrophy, Spinal/physiopathology ; Oligonucleotides/economics ; Surveys and Questionnaires ; Time Factors ; Treatment Outcome ; Young Adult
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Lancet Neurol. 2020 Apr;19(4):317-325. (PMID: 32199097)
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Orphanet J Rare Dis. 2017 Nov 28;12(1):175. (PMID: 29183396)
Muscle Nerve. 2021 Mar;63(3):311-319. (PMID: 33184859)
Muscle Nerve. 2020 Feb;61(2):222-226. (PMID: 31773738)
Neuromuscul Disord. 2018 Mar;28(3):197-207. (PMID: 29305137)
Contributed Indexing:: Keywords: SMA; burden; nusinersen; patient reported outcome; spinal muscular atrophy; unmet need
Substance Nomenclature:: 0 (Oligonucleotides)
5Z9SP3X666 (nusinersen)
Entry Date(s):: Date Created: 20201113 Date Completed: 20210401 Latest Revision: 20210401
Update Code:: 20240105
PubMed Central ID:: PMC7986200
DOI:: 10.1002/mus.27116
PMID:: 33184859
: Czasopismo naukowe

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Background: This study aimed to examine the early experience of nusinersen for spinal muscular atrophy (SMA) from the patient and caregiver perspective.
Methods: A 54-item online survey was administered to adult patients and caregivers of pediatric patients diagnosed with SMA.
Results: Overall, respondents (56 patients and 45 caregivers) were satisfied with nusinersen. Satisfaction was highest on changes in energy, stamina, and motor function and lowest on treatment administration and overall time commitment. Differences were noted for treatment effect sustained over time as reported by adult patients vs caregivers reporting on behalf of pediatric patients. Respondents reported insurance approval as a key barrier to access, particularly among adult patients.
Conclusions: Despite therapeutic advances, there remain significant unmet needs for SMA. Challenges with administration and barriers to access potentially limit the number of patients treated or delay treatment. Continued efforts are needed to develop more treatment options and to improve access to treatments.
(© 2020 The Authors. Muscle & Nerve published by Wiley Periodicals LLC.)

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