Navigating the Rehabilitation and Orthopedic Complications of SMA-Video Presentation

Video Transcription

Video Summary

Combination therapy is not currently used in the treatment of spinal muscular atrophy (SMA) and there is no evidence on the efficacy of combining different therapies. The current approved therapies for SMA include Nusinersen (Spinraza), Onasemnogene Abeparvovec-xioi (Zolgensma), and Risdiplam. Nusinersen is administered intrathecally and is indicated for SMA patients of all types. Onasemnogene Abeparvovec-xioi is administered intravenously and is indicated for infants with SMA. Risdiplam is an oral medication and is indicated for children and adults above two months of age. Clinical trials have shown that these therapies can improve motor milestones and survival rates in SMA patients. The therapies primarily work by increasing SMN protein levels, which is crucial for preventing disease progression. However, there is ongoing research on combination therapies for SMA, particularly the use of myostatin inhibitors alongside current treatments. These combination therapies are being investigated for their potential to enhance muscle strength and improve overall function in SMA patients.

Keywords

Combination therapy

spinal muscular atrophy

SMA

Nusinersen

Onasemnogene Abeparvovec-xioi

Risdiplam

motor milestones

survival rates

SMN protein levels

myostatin inhibitors