How Salbutamol May Help Patients with Spinal Muscular Atrophy: New Insights

How Salbutamol May Help Patients with Spinal Muscular Atrophy: New Insights

The vast majority of spinal muscular atrophy (SMA) cases are caused by a mutation in the survival motor neuron 1 (SMN1) gene, which leads to abnormally low levels of SMN1 gene products.1 Many attempts at therapy have focused on how to increase SMN levels in SMA...
Alternate Nusinersen Administration Strategies

Alternate Nusinersen Administration Strategies

In the pivotal clinical trials that support FDA approval, nusinersen was administered via intrathecal injection. However, this approach requires that patients receive multiple intrathecal injections separated in time by weeks. During each infusion, the clinician must...
Nusinersen

Nusinersen

Nusinersen (Spinraza) is the first FDA-approved, disease-modifying therapy for the treatment of spinal muscular atrophy (SMA). Nusinersen is an antisense oligonucleotide that binds to a sequence downstream of exon 7 in the SMN2 gene. The oligonucleotide blocks the...