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

Abstract

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.

Ultrasound‐guided transforaminal approach for nusinersen delivery in adult spinal muscle atrophy patients with challenging access

“…Since several factors may influence the patient's decision for treatment initiation or maintenance, 19 it is important to minimize the factors that cause the patient's discomfort. The only patient that preferred CBCT‐guidance over US‐guidance was the one in whom the prominent vein was inadvertently accessed, an event previously described in CBCT‐guided injections 8 .…”

Assessment of Bulbar Function in Adult Patients with 5q-SMA Type 2 and 3 under Treatment with Nusinersen

“…Additionally, data on swallowing ability under risdiplam in infants imply preserved swallowing function and oral feeding ability throughout the first 12 months of therapy, indicating a relevant therapeutic effect of this gene-based therapy [ 28 ]. In an online survey on satisfaction with early nusinersen therapy, adult and pediatric SMA patients rated the effect of nusinersen on bulbar symptoms such as eating and talking similarly as satisfactory by about 65% [ 29 ].…”

Development of the SMA independence scale–upper limb module (SMAIS–ULM): A novel scale for individuals with Type 2 and non-ambulant Type 3 SMA

“…Individuals aged ≥12 years completed the self-report form and caregivers for all individuals aged 2-25 years old completed the caregiver-report form. Secondly, a survey for individuals with SMA and caregivers (US unmet need survey) was administered online at a single time point to individuals aged ≥18 years and caregivers of individuals aged <18 years [71]. The survey aimed to capture opinions, preference, and experiences with existing SMA treatments.…”