– Phase 3 trial is a randomized, double-blind, placebo-controlled trial of apitegromab as add-on to background SMN therapy in non-ambulatory Type 2/3 SMA – Trial design is informed by the ...

SAPPHIRE is an ongoing randomized, double-blind, placebo-controlled, phase 3 clinical trial evaluating the safety and efficacy of apitegromab in non-ambulatory patients with Types 2 and 3 SMA who ...

Type 2 would never be able to walk. And so they will remain non-ambulatory, meaning that they will re-- require wheelchair and other transportations from one place to another. Type 3 and 4 are ...

Scholar Rock reported that at 48 months, more than 90% of the non-ambulatory patients enrolled in the study remained on apitegromab treatment and there was a sustained motor function improvement ...

Children with non-ambulatory type I SMA developed scoliosis at a similar but slightly higher rate than those with type II (90% and 88%, respectively) (I/II, P=.02).

The Bottom Line. Researchers collected completed questionnaires from 48 parents of children with varying levels of SMA. Scores were lower for caregivers of children who were non-ambulatory and ...

The efficacy data from the Phase 2 TOPAZ trial extension period showed sustained and continued improvement with apitegromab for non-ambulatory patients with Types 2 and 3 SMA receiving an SMN therapy.

Wolfe A, Scoto M, Milev E, et al. Longitudinal changes in respiratory and upper limb function in a pediatric type III spinal muscular atrophy cohort after loss of ambulation. Muscle Nerve.

van Bruggen HW, van den Engel-Hoek L, van der Pol WL, et al. Impaired mandibular function in spinal muscular atrophy type II: need for early recognition. J Child Neurol . 2011. doi:10.1177 ...

On 8 August 2024, as part of its second quarter (Q2) financial results and business progress reporting, Scholar Rock disclosed new positive data from its Phase II TOPAZ trial (NCT03921528 ...