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

Autor: Rupali Naik, Josh M. Noone, Stacy Dixon, William Arnold, J. Daniel Buchenberger, Sarah M. Whitmire, Er Chen, Rosalina Mills
Rok vydání: 2020
Předmět:
Male
0301 basic medicine
Time Factors
Physiology
Oligonucleotides
030105 genetics & heredity
Insurance Coverage
burden
0302 clinical medicine
Surveys and Questionnaires
Activities of Daily Living
Medicine
SMA
Child
Injections
Spinal
spinal muscular atrophy
Clinical Research Article
nusinersen
Treatment options
Middle Aged
unmet need
Treatment Outcome
Caregivers
Patient Satisfaction
Child
Preschool
Female
Patient-reported outcome
Nusinersen
Adult
medicine.medical_specialty
Adolescent
Unmet needs
Muscular Atrophy
Spinal
Young Adult
03 medical and health sciences
Cellular and Molecular Neuroscience
Physiology (medical)
Humans
Treatment effect
Clinical Research Articles
Insurance
Health
Adult patients
business.industry
Infant
Spinal muscular atrophy
medicine.disease
patient reported outcome
Physical therapy
Neurology (clinical)
Health Expenditures
business
030217 neurology & neurosurgery
Zdroj: Muscle & Nerve
ISSN: 1097-4598
0148-639X
DOI: 10.1002/mus.27116
Popis: 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.
Databáze: OpenAIRE
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